Charles Gersbach
John W. Strohbehn Distinguished Professor of Biomedical Engineering
Current Research Interests
Gene therapy, genomics and epigenomics, biomolecular and cellular engineering, regenerative medicine, and synthetic biology.
Current Appointments & Affiliations
- John W. Strohbehn Distinguished Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2021
- Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2021
- Associate Professor of Surgery, Surgery, Surgical Sciences, Surgery 2019
- Associate Professor in Orthopaedic Surgery, Orthopaedic Surgery, Clinical Science Departments 2015
- Associate Professor in Cell Biology, Cell Biology, Basic Science Departments 2020
- Member of the Duke Cancer Institute, Duke Cancer Institute, Institutes and Centers 2013
- Associate of the Duke Initiative for Science & Society, Duke Science & Society, Initiatives 2017
- Core Faculty in Innovation & Entrepreneurship, Duke Innovation & Entrepreneurship, Initiatives 2019
- Affiliate of the Duke Regeneration Center, Regeneration Next Initiative, Basic Science Departments 2021
Contact Information
- 2123 CIEMAS, Durham, NC 27708
- Box 90281, 136 Hudson Hall, Durham, NC 27708
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charles.gersbach@duke.edu
(919) 613-2147
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Google Scholar
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http://gersbach.bme.duke.edu/
- Background
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Education, Training, & Certifications
- Ph.D., Georgia Institute of Technology 2006
- B.S., Georgia Institute of Technology 2001
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Previous Appointments & Affiliations
- Associate Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2015 - 2020
- Rooney Family Associate Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2016 - 2020
- Assistant Professor of Orthopaedic Surgery, Orthopaedic Surgery, Clinical Science Departments 2013 - 2015
- Assistant Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2009 - 2015
- Investigator in the Institute for Genome Sciences & Policy, Institutes and Centers, School of Medicine 2012 - 2014
- Assistant Research Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2009
- Recognition
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In the News
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DEC 13, 2022 -
JUN 28, 2021 -
FEB 5, 2021 School of Medicine -
DEC 2, 2020 School of Medicine -
OCT 1, 2019 Pratt School of Engineering -
SEP 24, 2019 Pratt School of Engineering -
APR 15, 2019 Pratt School of Engineering -
FEB 18, 2019 Pratt School of Engineering -
DEC 17, 2018 Duke Research Blog -
OCT 3, 2018 Duke Med Alumni News -
APR 27, 2018 -
APR 26, 2018 Pratt School of Engineering -
APR 9, 2018 -
NOV 29, 2017 -
APR 4, 2017 Pratt School of Engineering -
APR 3, 2017 The Scientist -
MAR 15, 2017 CNBC -
MAY 5, 2016 -
JAN 4, 2016 The New York Times -
JAN 4, 2016 BBC News -
DEC 31, 2015 -
OCT 27, 2015 -
MAY 26, 2015 -
APR 6, 2015 -
FEB 19, 2015 -
FEB 18, 2015 -
FEB 10, 2015
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Awards & Honors
- John W. Strohbehn Distinguished Professor of Biomedical Engineering . Duke University. 2021
- Stansell Family Distinguished Research Award . Pratt School of Engineering. 2019
- Allen Distinguished Investigator. Paul G. Allen Frontiers Group. 2017
- Fellow. American Institute for Medical and Biological Engineering. 2017
- Thomas Langford Lectureship Award. Duke University. 2016
- Outstanding New Investigator. American Society of Gene and Cell Therapy. 2014
- Capers and Marion McDonald Teaching and Research Award. Duke University. 2013
- Faculty Early Career Development (CAREER) Program. National Science Foundation. 2012
- Rising Star Award. Society for Physical Regulation in Biology and Medicine. 2012
- Basil O’Connor Starter Scholar Research Award. March of Dimes Foundation. 2011
- NIH Director’s New Innovator Award. National Institutes of Health. 2011
- Ralph E. Powe Junior Faculty Enhancement Award. Oak Ridge Associated Universities. 2011
- Hartwell Individual Biomedical Research Award. The Hartwell Foundation. 2010
- Research
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Selected Grants
- University Training Program in Biomolecular and Tissue Engineering awarded by National Institutes of Health 1994 - 2027
- Training Program in Developmental and Stem Cell Biology awarded by National Institutes of Health 2001 - 2027
- Oxidative stress mechanisms regulating gamma-globin gene transcription in sickle cell disease awarded by National Institutes of Health 2022 - 2026
- Cell and Molecular Biology Training Program awarded by National Institutes of Health 2021 - 2026
- High-Throughput Functional Annotation of Gene Regulatory Elements and Variants Critical to Complex Cellular Phenotypes awarded by National Institutes of Health 2021 - 2026
- Engineering Human Heart Tissues with Polyploid Cardiomyocytes awarded by National Institutes of Health 2022 - 2026
- Beyond GWAS: High Throughput Functional Genomics & Epigenome Editing to Elucidate the Effects of Genetic Associations for Schizophrenia awarded by National Institutes of Health 2021 - 2026
- Multi-omics functional analysis of non-coding regulatory genome for genomic medicine awarded by National Institutes of Health 2020 - 2025
- Genetic and Genomics Training Grant awarded by National Institutes of Health 2020 - 2025
- Uncovering novel gene regulatory mechanisms underlying glucocorticoid response phenotypes through targeted mutagenesis of an essential transcription factor awarded by National Institutes of Health 2022 - 2025
- Lewy body neuropathologies and SNCA gene: variants expression and splicing awarded by National Institutes of Health 2020 - 2025
- Genome Editing for the Treatment of Neurofibromatosis Type I awarded by Gilbert Family Foundation 2022 - 2024
- Exploring the Use of Gene Therapies to Treat Neurofibromatosis Type I awarded by National Institutes of Health 2022 - 2024
- Engineering epigenome editing tools for sustained reactivation of maternal PWS genes awarded by Foundation for Prader-Willi Research 2020 - 2024
- Epigenome editing of the Prader-Willi syndrome imprinted domain with CRISPR/Cas9 awarded by National Institutes of Health 2021 - 2024
- Mechanotransduction in Meniscus Health and Repair awarded by National Institutes of Health 2019 - 2023
- Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes awarded by National Institutes of Health 2019 - 2023
- Epigenomic Reprogramming in Patient Derived Models of Colorectal Cancer awarded by National Institutes of Health 2018 - 2023
- Evolving High Potency AAV Vectors for Neuromuscular Genome Editing awarded by National Institutes of Health 2018 - 2023
- The function and regulation of the novel pregnancy-specific hexokinase HKDC1 awarded by University of Illinois at Chicago 2017 - 2023
- Inductive repair of human kidney tissues by cell type-specific sensing and reprogramming awarded by University of Michigan 2021 - 2023
- The Duke FUNCTION Center: Pioneering the comprehensive identification of combinatorial noncoding causes of disease awarded by National Institutes of Health 2020 - 2023
- Epigenome Editing Technologies for Treating Diverse Disease awarded by National Institutes of Health 2019 - 2023
- Preparing Genetic Counselors for Genomic Medicine Research awarded by National Institutes of Health 2017 - 2023
- Quantifying the genetic diversity of human regulatory element activity awarded by National Institutes of Health 2019 - 2023
- Postdoctoral Training in Genomic Medicine Research awarded by National Institutes of Health 2017 - 2023
- IRES Track 1 IRTG Engaged in Dissecting and Reengineering the Regulatory Genome awarded by National Science Foundation 2019 - 2023
- Thwarting Influenza with RNA-powered Modulators (ThIRM) awarded by Georgia Institute of Technology 2019 - 2023
- The Branched Chain Ketoacid Dehydrogenase Kinase-Phosphatase System as a New Regulatory Node in Myocardial Fuel Section awarded by National Institutes of Health 2018 - 2023
- Epigenome Editing Therapy to Treat Mouse Models of Prader-Willi Syndrome awarded by Levo Therapeutics, Inc 2020 - 2022
- Editing the Kidney Epigenome awarded by University of Washington 2021 - 2022
- Dissecting Mechanisms by which p53 Suppresses Transformation and Radiation Carcinogenesis awarded by National Institutes of Health 2020 - 2022
- EFRI CEE : Engineering Technologies to Determine Causal Relationships Between Chromatin Structure and Gene Regulation awarded by National Science Foundation 2018 - 2022
- To support research on the development of CRISPR-based epigenome editing tools to refine genome wide association studies awarded by Open Philanthropy Project 2018 - 2022
- An organotypic model recapitulating colon cancer microenvironment and metastasis awarded by University of Texas Southwestern Medical Center 2017 - 2022
- Regulatory Mechanisms of CD4+ T Cell Differentiation awarded by National Institutes of Health 2017 - 2022
- Systemic Inflammation in Microphysiological Models of Muscle and Vascular Disease awarded by National Institutes of Health 2017 - 2022
- REU Site for Meeting the Grand Challenges in Engineering awarded by National Science Foundation 2017 - 2022
- In Vivo Epigenome Editing with CRISPR-Based Histone Acetyltransferase Transgenic Mice awarded by National Institutes of Health 2016 - 2022
- Investigating Autophagy in GSD-Ia awarded by National Institutes of Health 2020 - 2021
- Bioinformatics and Computational Biology Training Program awarded by National Institutes of Health 2005 - 2021
- Epigenome Editing Technologies to Control Diverse Biological Functions awarded by Allen Institute for Brain Science 2017 - 2021
- Enhancing Neuronal Reprogramming with Epigenome Editing awarded by National Institutes of Health 2018 - 2021
- CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular Dystrophy awarded by National Institutes of Health 2016 - 2021
- Genome Editing For Duchenne Muscular Dystrophy awarded by Sarepta Therapeutics, Inc. 2017 - 2021
- Investigating Autophagy in GSD-Ia awarded by National Institutes of Health 2015 - 2020
- Genetics Training Grant awarded by National Institutes of Health 1979 - 2020
- Treating Duchenne Cardiomyopathy in the Mouse Model by Gene Repair awarded by University of Missouri 2016 - 2020
- Identification and application of cis-regulatory enhancer elements for muscle stem cell function and muscle regeneration in aging awarded by American Federation for Aging Research 2019 - 2020
- Combinatorial Biomimetic Systems for High-Throughput Manipulation of Genome-Edited Dystrophic Muscle Stem Cells awarded by Cornell University 2018 - 2020
- Collaborative Research: Adapting Cas9 Protein from CRISPR as a Structural Unit for Molecular Assembly awarded by National Science Foundation 2017 - 2020
- Functional characterization of regulatory variants associated with maternal hyperglycemia awarded by National Institutes of Health 2017 - 2020
- Designing a next generation vector for CRISPR/Cas9-mediated gene editing to treat Duchenne muscular dystrophy awarded by North Carolina Biotechnology Center 2018 - 2020
- Developing Brain-penetrant G9a/GLP Inhibitors for Treating Prader-Willi Syndrome awarded by Levo Therapeutics, Inc 2018 - 2020
- NONHUMAN PRIMATE TESTING CENTER FOR EVALUATION OF SOMATIC CELL GENOME EDITING TOOLS awarded by University of California - Davis 2019 - 2020
- Development of AAV-Based Curative Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration awarded by North Carolina Biotechnology Center 2017 - 2019
- Enhancing Neuronal Reprogramming with Epigenome Editing awarded by National Institutes of Health 2017 - 2019
- Non-viral delivery of CRISPR/Cas9 for targeted gene replacement awarded by National Institutes of Health 2018 - 2019
- Novel Approaches for Correcting Respiratory Insufficiency in Pompe Disease awarded by National Institutes of Health 2014 - 2019
- Reprogrammed autologous T cells to promote transplant tolerance awarded by Element Genomics, Inc. 2018 - 2019
- Systemically administrered EGRFvIII-targeted bispecific antibody as an immunotherapeutic for glioblastoma. awarded by National Institutes of Health 2015 - 2019
- Defining the Mechanisms of Gene Regulation and Drug Resistance in Cancer awarded by Thorek Memorial Foundation 2017 - 2019
- Decoding and Reprogramming the Corticosteroid Transcriptional Regulatory Network awarded by National Institutes of Health 2015 - 2018
- Trans-regulatory mechanisms of glucocorticoid-mediated transcriptional repression awarded by National Institutes of Health 2016 - 2018
- Genome engineering for Duchenne muscular dystrophy awarded by American Heart Association 2017 - 2018
- Engineering Novel Genome Engineering Systems awarded by Locus Biosciences, Inc. 2016 - 2018
- Circulatory system and integrated muscle tissue for drug and tissue toxicity awarded by National Institutes of Health 2012 - 2018
- Engineering Targeted Epigenetic Modifiers for Precise Control of Gene Regulation awarded by National Institutes of Health 2013 - 2018
- A Platform Technology for High-Throughput Screening of Gene Regulatory Elements awarded by Element Genomics, Inc. 2016 - 2017
- Functional Tissue Engineering of Cartilage Using Induced Pluripotent Stem Cells awarded by National Institutes of Health 2015 - 2017
- MD140071 Multiplex CRISPR/Cas9-Based Genome Engineering for the Genetic Correction of Duchenne Muscular Dystrophy awarded by United States Army Medical Research and Materiel Command 2015 - 2017
- CRISPR/Cas9-based genome editing in mouse models of Duchenne muscular dystrophy awarded by Hartwell Foundation 2015 - 2017
- Scaffold-Mediated Gene Delivery for Engineering of Osteochondral Tissues awarded by National Institutes of Health 2015 - 2017
- Gertude B. Elion Mentored Medical Research Award awarded by Triangle Community Foundation 2016 - 2017
- Genome Editing of Stem Cells for Analysis of Osteoarthritis Causal Variants awarded by National Institutes of Health 2014 - 2017
- CAREER: Photoregulated Gene Expression for Spatiotemporal Control of Morphogenesis awarded by National Science Foundation 2012 - 2017
- Engineering Morphogenetic Factors for Enhanced Genetic Reprogramming awarded by National Institutes of Health 2011 - 2016
- Engineering New Biological Therapies for Arthritis awarded by Arthritis Foundation 2014 - 2015
- Live-Animal Micro-CT System awarded by National Institutes of Health 2012 - 2014
- Spatially Controlled Gene Delivery of Morphogenetic Factors from Woven Scaffolds awarded by National Institutes of Health 2011 - 2014
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External Relationships
- Editas Medicine
- Element Genomics (UCB Pharma)
- Iveric Bio
- Levo Therapeutics, Inc.
- Locus Biosciences
- Sarepta Therapeutics
- Tune Therapeutics
- Publications & Artistic Works
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Selected Publications
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Books
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Abdeen, Amr A., Brian D. Cosgrove, Charles A. Gersbach, and Krishanu Saha. Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. Vol. 23, 2021. https://doi.org/10.1146/annurev-bioeng-122019-121602.Full Text
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Holtzman, Liad, and Charles A. Gersbach. Editing the Epigenome: Reshaping the Genomic Landscape. Vol. 19, 2018. https://doi.org/10.1146/annurev-genom-083117-021632.Full Text
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Academic Articles
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Yan, Ruorong, Valentina Cigliola, Kelsey A. Oonk, Zachary Petrover, Sophia DeLuca, David W. Wolfson, Andrew Vekstein, et al. “An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair.” Cell Stem Cell 30, no. 1 (January 5, 2023): 96-111.e6. https://doi.org/10.1016/j.stem.2022.11.012.Full Text Link to Item
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Gonzalez, Trevor J., Katherine E. Simon, Leo O. Blondel, Marco M. Fanous, Angela L. Roger, Maribel Santiago Maysonet, Garth W. Devlin, et al. “Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.” Nat Commun 13, no. 1 (October 10, 2022): 5947. https://doi.org/10.1038/s41467-022-33745-4.Full Text Link to Item
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Beyersdorf, Jared P., Swapnil Bawage, Nahid Iglesias, Hannah E. Peck, Ryan A. Hobbs, Jay A. Wroe, Chiara Zurla, Charles A. Gersbach, and Philip J. Santangelo. “Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators.” Acs Nano 16, no. 4 (April 2022): 5660–71. https://doi.org/10.1021/acsnano.1c10631.Full Text
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Pickar-Oliver, Adrian, Veronica Gough, Joel D. Bohning, Siyan Liu, Jacqueline N. Robinson-Hamm, Heather Daniels, William H. Majoros, Garth Devlin, Aravind Asokan, and Charles A. Gersbach. “Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.” Mol Ther 29, no. 11 (November 3, 2021): 3243–57. https://doi.org/10.1016/j.ymthe.2021.09.003.Full Text Link to Item
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Hakim, Chady H., Sandeep R. P. Kumar, Dennis O. Pérez-López, Nalinda B. Wasala, Dong Zhang, Yongping Yue, James Teixeira, et al. “Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.” Nature Communications 12, no. 1 (November 2021): 6769. https://doi.org/10.1038/s41467-021-26830-7.Full Text
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Wang, Lihua, Ergang Wang, Jorge Prado Balcazar, Zhenzhen Wu, Kun Xiang, Yi Wang, Qiang Huang, et al. “Chromatin Remodeling of Colorectal Cancer Liver Metastasis is Mediated by an HGF-PU.1-DPP4 Axis.” Adv Sci (Weinh) 8, no. 19 (October 2021): e2004673. https://doi.org/10.1002/advs.202004673.Full Text Link to Item
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Gemberling, Matthew P., Keith Siklenka, Erica Rodriguez, Katherine R. Tonn-Eisinger, Alejandro Barrera, Fang Liu, Ariel Kantor, et al. “Transgenic mice for in vivo epigenome editing with CRISPR-based systems.” Nat Methods 18, no. 8 (August 2021): 965–74. https://doi.org/10.1038/s41592-021-01207-2.Full Text Link to Item
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Bodle, Josephine C., and Charles A. Gersbach. “CRISPR Clocks: The Times They Are a-Changin'.” The Crispr Journal 4, no. 2 (April 2021): 160–63. https://doi.org/10.1089/crispr.2021.29123.ger.Full Text
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Saha, Krishanu, Erik J. Sontheimer, P. J. Brooks, Melinda R. Dwinell, Charles A. Gersbach, David R. Liu, Stephen A. Murray, et al. “The NIH Somatic Cell Genome Editing program.” Nature 592, no. 7853 (April 2021): 195–204. https://doi.org/10.1038/s41586-021-03191-1.Full Text Link to Item
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Seo, Jungkyun, D Dewran Koçak, Luke C. Bartelt, Courtney A. Williams, Alejandro Barrera, Charles A. Gersbach, and Timothy E. Reddy. “AP-1 subunits converge promiscuously at enhancers to potentiate transcription.” Genome Res 31, no. 4 (April 2021): 538–50. https://doi.org/10.1101/gr.267898.120.Full Text Link to Item
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Walejko, Jacquelyn M., Bridgette A. Christopher, Scott B. Crown, Guo-Fang Zhang, Adrian Pickar-Oliver, Takeshi Yoneshiro, Matthew W. Foster, et al. “Branched-chain α-ketoacids are preferentially reaminated and activate protein synthesis in the heart.” Nat Commun 12, no. 1 (March 15, 2021): 1680. https://doi.org/10.1038/s41467-021-21962-2.Full Text Link to Item
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Kwon, Jennifer B., Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, and Charles A. Gersbach. “In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy.” Mol Ther Methods Clin Dev 19 (December 11, 2020): 320–29. https://doi.org/10.1016/j.omtm.2020.09.016.Full Text Link to Item
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Black, Joshua B., Sean R. McCutcheon, Shataakshi Dube, Alejandro Barrera, Tyler S. Klann, Grayson A. Rice, Shaunak S. Adkar, Scott H. Soderling, Timothy E. Reddy, and Charles A. Gersbach. “Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens.” Cell Rep 33, no. 9 (December 1, 2020): 108460. https://doi.org/10.1016/j.celrep.2020.108460.Full Text Link to Item
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Bulaklak, Karen, and Charles A. Gersbach. “The once and future gene therapy.” Nature Communications 11, no. 1 (November 2020): 5820. https://doi.org/10.1038/s41467-020-19505-2.Full Text
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Carullo, Nancy V. N., Robert A. Phillips Iii, Rhiana C. Simon, Salomon A Roman Soto, Jenna E. Hinds, Aaron J. Salisbury, Jasmin S. Revanna, et al. “Enhancer RNAs predict enhancer-gene regulatory links and are critical for enhancer function in neuronal systems.” Nucleic Acids Research 48, no. 17 (September 2020): 9550–70. https://doi.org/10.1093/nar/gkaa671.Full Text
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Cosgrove, Brian D., and Charles A. Gersbach. “Unwinding the Role of FACT in Cas9-based Genome Editing.” Molecular Cell 79, no. 3 (August 2020): 365–67. https://doi.org/10.1016/j.molcel.2020.07.016.Full Text
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Mao, Mao, Chun-Chi Chang, Adrian Pickar-Oliver, Lisa D. Cervia, Liangli Wang, Jing Ji, Paloma B. Liton, Charles A. Gersbach, and Fan Yuan. “Redirecting Vesicular Transport to Improve Nonviral Delivery of Molecular Cargo.” Adv Biosyst 4, no. 8 (August 2020): e2000059. https://doi.org/10.1002/adbi.202000059.Full Text Link to Item
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Gough, Veronica, and Charles A. Gersbach. “Immunity to Cas9 as an Obstacle to Persistent Genome Editing.” Molecular Therapy : The Journal of the American Society of Gene Therapy 28, no. 6 (June 2020): 1389–91. https://doi.org/10.1016/j.ymthe.2020.05.007.Full Text
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Kwon, Jennifer B., Ashish Vankara, Adarsh R. Ettyreddy, Joel D. Bohning, and Charles A. Gersbach. “Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators.” Stem Cell Reports 14, no. 5 (May 2020): 755–69. https://doi.org/10.1016/j.stemcr.2020.03.026.Full Text
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Dicks, Amanda, Chia-Lung Wu, Nancy Steward, Shaunak S. Adkar, Charles A. Gersbach, and Farshid Guilak. “Prospective isolation of chondroprogenitors from human iPSCs based on cell surface markers identified using a CRISPR-Cas9-generated reporter.” Stem Cell Research & Therapy 11, no. 1 (February 2020): 66. https://doi.org/10.1186/s13287-020-01597-8.Full Text
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Huang, Jianguo, Mark Chen, Eric S. Xu, Lixia Luo, Yan Ma, Wesley Huang, Warren Floyd, et al. “Genome-wide CRISPR Screen to Identify Genes that Suppress Transformation in the Presence of Endogenous KrasG12D.” Sci Rep 9, no. 1 (November 20, 2019): 17220. https://doi.org/10.1038/s41598-019-53572-w.Full Text Open Access Copy Link to Item
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Evans, Brian C., R Brock Fletcher, Kameron V. Kilchrist, Eric A. Dailing, Alvin J. Mukalel, Juan M. Colazo, Matthew Oliver, et al. “An anionic, endosome-escaping polymer to potentiate intracellular delivery of cationic peptides, biomacromolecules, and nanoparticles.” Nature Communications 10, no. 1 (November 2019): 5012. https://doi.org/10.1038/s41467-019-12906-y.Full Text
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Kwon, Jennifer B., and Charles A. Gersbach. “Jumping at the chance for precise DNA integration.” Nature Biotechnology 37, no. 9 (September 2019): 1004–6. https://doi.org/10.1038/s41587-019-0210-3.Full Text
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Nance, Michael E., Ruicheng Shi, Chady H. Hakim, Nalinda B. Wasala, Yongping Yue, Xiufang Pan, Tracy Zhang, et al. “AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.” Molecular Therapy : The Journal of the American Society of Gene Therapy 27, no. 9 (September 2019): 1568–85. https://doi.org/10.1016/j.ymthe.2019.06.012.Full Text
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Pickar-Oliver, Adrian, and Charles A. Gersbach. “The next generation of CRISPR-Cas technologies and applications.” Nature Reviews. Molecular Cell Biology 20, no. 8 (August 2019): 490–507. https://doi.org/10.1038/s41580-019-0131-5.Full Text
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Kocak, D Dewran, Eric A. Josephs, Vidit Bhandarkar, Shaunak S. Adkar, Jennifer B. Kwon, and Charles A. Gersbach. “Increasing the specificity of CRISPR systems with engineered RNA secondary structures.” Nature Biotechnology 37, no. 6 (June 2019): 657–66. https://doi.org/10.1038/s41587-019-0095-1.Full Text
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Nelson, Christopher E., Yaoying Wu, Matthew P. Gemberling, Matthew L. Oliver, Matthew A. Waller, Joel D. Bohning, Jacqueline N. Robinson-Hamm, et al. “Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.” Nat Med 25, no. 3 (March 2019): 427–32. https://doi.org/10.1038/s41591-019-0344-3.Full Text Link to Item
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Chen, Liang-Fu, Yen Ting Lin, David A. Gallegos, Mariah F. Hazlett, Mariana Gómez-Schiavon, Marty G. Yang, Breanna Kalmeta, et al. “Enhancer Histone Acetylation Modulates Transcriptional Bursting Dynamics of Neuronal Activity-Inducible Genes.” Cell Rep 26, no. 5 (January 29, 2019): 1174-1188.e5. https://doi.org/10.1016/j.celrep.2019.01.032.Full Text Link to Item
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Adkar, Shaunak S., Chia-Lung Wu, Vincent P. Willard, Amanda Dicks, Adarsh Ettyreddy, Nancy Steward, Nidhi Bhutani, Charles A. Gersbach, and Farshid Guilak. “Step-Wise Chondrogenesis of Human Induced Pluripotent Stem Cells and Purification Via a Reporter Allele Generated by CRISPR-Cas9 Genome Editing.” Stem Cells (Dayton, Ohio) 37, no. 1 (January 2019): 65–76. https://doi.org/10.1002/stem.2931.Full Text
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Hakim, Chady H., Nalinda B. Wasala, Christopher E. Nelson, Lakmini P. Wasala, Yongping Yue, Jacqueline A. Louderman, Thais B. Lessa, et al. “AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.” Jci Insight 3, no. 23 (December 2018): 124297. https://doi.org/10.1172/jci.insight.124297.Full Text
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Black, Joshua B., and Charles A. Gersbach. “Synthetic transcription factors for cell fate reprogramming.” Current Opinion in Genetics & Development 52 (October 2018): 13–21. https://doi.org/10.1016/j.gde.2018.05.001.Full Text
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Huynh, Nguyen P. T., Jonathan M. Brunger, Catherine C. Gloss, Franklin T. Moutos, Charles A. Gersbach, and Farshid Guilak. “Genetic Engineering of Mesenchymal Stem Cells for Differential Matrix Deposition on 3D Woven Scaffolds.” Tissue Engineering. Part A 24, no. 19–20 (October 2018): 1531–44. https://doi.org/10.1089/ten.tea.2017.0510.Full Text
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Gersbach, C. A. “Gene delivery and biomedical engineering.” Current Opinion in Biomedical Engineering 7 (September 1, 2018): iii–v. https://doi.org/10.1016/j.cobme.2018.11.003.Full Text
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McDowell, Ian C., Alejandro Barrera, Anthony M. D’Ippolito, Christopher M. Vockley, Linda K. Hong, Sarah M. Leichter, Luke C. Bartelt, et al. “Glucocorticoid receptor recruits to enhancers and drives activation by motif-directed binding.” Genome Res 28, no. 9 (September 2018): 1272–84. https://doi.org/10.1101/gr.233346.117.Full Text Link to Item
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D’Ippolito, Anthony M., Ian C. McDowell, Alejandro Barrera, Linda K. Hong, Sarah M. Leichter, Luke C. Bartelt, Christopher M. Vockley, et al. “Pre-established Chromatin Interactions Mediate the Genomic Response to Glucocorticoids.” Cell Syst 7, no. 2 (August 22, 2018): 146-160.e7. https://doi.org/10.1016/j.cels.2018.06.007.Full Text Link to Item
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Klann, Tyler S., Joshua B. Black, and Charles A. Gersbach. “CRISPR-based methods for high-throughput annotation of regulatory DNA.” Current Opinion in Biotechnology 52 (August 2018): 32–41. https://doi.org/10.1016/j.copbio.2018.02.004.Full Text
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Kocak, D Dewran, and Charles A. Gersbach. “From CRISPR scissors to virus sensors.” Nature 557, no. 7704 (May 2018): 168–69. https://doi.org/10.1038/d41586-018-04975-8.Full Text
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Thakore, Pratiksha I., Jennifer B. Kwon, Christopher E. Nelson, Douglas C. Rouse, Matthew P. Gemberling, Matthew L. Oliver, and Charles A. Gersbach. “RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors.” Nature Communications 9, no. 1 (April 2018): 1674. https://doi.org/10.1038/s41467-018-04048-4.Full Text
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Gersbach, Charles A., and Rodolphe Barrangou. “Pulling the genome in opposite directions to dissect gene networks.” Genome Biology 19, no. 1 (March 2018): 42. https://doi.org/10.1186/s13059-018-1425-1.Full Text
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Gemberling, Matthew, and Charles A. Gersbach. “Boosting, Not Breaking: CRISPR Activators Treat Disease Models.” Molecular Therapy : The Journal of the American Society of Gene Therapy 26, no. 2 (February 2018): 334–36. https://doi.org/10.1016/j.ymthe.2018.01.004.Full Text
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Pickar, Adrian K., and Charles A. Gersbach. “Gene therapies for hemophilia hit the mark in clinical trials.” Nature Medicine 24, no. 2 (February 2018): 121–22. https://doi.org/10.1038/nm.4492.Full Text
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Klann, Tyler S., Gregory E. Crawford, Timothy E. Reddy, and Charles A. Gersbach. “Screening Regulatory Element Function with CRISPR/Cas9-based Epigenome Editing.” Methods Mol Biol 1767 (2018): 447–80. https://doi.org/10.1007/978-1-4939-7774-1_25.Full Text Link to Item
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Gersbach, C. A. “Editorial Overview: Synthetic biology and biomedical engineering.” Current Opinion in Biomedical Engineering 4 (December 1, 2017): vi–vii. https://doi.org/10.1016/j.cobme.2017.12.005.Full Text
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Manandhar, Dinesh, Lingyun Song, Ami Kabadi, Jennifer B. Kwon, Lee E. Edsall, Melanie Ehrlich, Koji Tsumagari, Charles A. Gersbach, Gregory E. Crawford, and Raluca Gordân. “Incomplete MyoD-induced transdifferentiation is associated with chromatin remodeling deficiencies.” Nucleic Acids Res 45, no. 20 (November 16, 2017): 11684–99. https://doi.org/10.1093/nar/gkx773.Full Text Link to Item
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Nelson, Christopher E., Jacqueline N. Robinson-Hamm, and Charles A. Gersbach. “Genome engineering: a new approach to gene therapy for neuromuscular disorders.” Nature Reviews. Neurology 13, no. 11 (November 2017): 647–61. https://doi.org/10.1038/nrneurol.2017.126.Full Text
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Pathak, Gopal P., Jessica I. Spiltoir, Camilla Höglund, Lauren R. Polstein, Sari Heine-Koskinen, Charles A. Gersbach, Jari Rossi, and Chandra L. Tucker. “Bidirectional approaches for optogenetic regulation of gene expression in mammalian cells using Arabidopsis cryptochrome 2.” Nucleic Acids Research 45, no. 20 (November 2017): e167. https://doi.org/10.1093/nar/gkx260.Full Text
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Polstein, Lauren R., Mark Juhas, Gabi Hanna, Nenad Bursac, and Charles A. Gersbach. “An Engineered Optogenetic Switch for Spatiotemporal Control of Gene Expression, Cell Differentiation, and Tissue Morphogenesis.” Acs Synthetic Biology 6, no. 11 (November 2017): 2003–13. https://doi.org/10.1021/acssynbio.7b00147.Full Text
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Adkar, Shaunak S., Jonathan M. Brunger, Vincent P. Willard, Chia-Lung Wu, Charles A. Gersbach, and Farshid Guilak. “Genome Engineering for Personalized Arthritis Therapeutics.” Trends in Molecular Medicine 23, no. 10 (October 2017): 917–31. https://doi.org/10.1016/j.molmed.2017.08.002.Full Text
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Farhang, Niloofar, Jonathan M. Brunger, Joshua D. Stover, Pratiksha I. Thakore, Brandon Lawrence, Farshid Guilak, Charles A. Gersbach, Lori A. Setton, and Robby D. Bowles. “* CRISPR-Based Epigenome Editing of Cytokine Receptors for the Promotion of Cell Survival and Tissue Deposition in Inflammatory Environments.” Tissue Engineering. Part A 23, no. 15–16 (August 2017): 738–49. https://doi.org/10.1089/ten.tea.2016.0441.Full Text
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Huang, Jianguo, Mark Chen, Melodi Javid Whitley, Hsuan-Cheng Kuo, Eric S. Xu, Andrea Walens, Yvonne M. Mowery, et al. “Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma.” Nat Commun 8 (July 10, 2017): 15999. https://doi.org/10.1038/ncomms15999.Full Text Link to Item
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Black, Joshua B., Pablo Perez-Pinera, and Charles A. Gersbach. “Mammalian Synthetic Biology: Engineering Biological Systems.” Annual Review of Biomedical Engineering 19 (June 2017): 249–77. https://doi.org/10.1146/annurev-bioeng-071516-044649.Full Text
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Klann, Tyler S., Joshua B. Black, Malathi Chellappan, Alexias Safi, Lingyun Song, Isaac B. Hilton, Gregory E. Crawford, Timothy E. Reddy, and Charles A. Gersbach. “CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome.” Nat Biotechnol 35, no. 6 (June 2017): 561–68. https://doi.org/10.1038/nbt.3853.Full Text Link to Item
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Brunger, Jonathan M., Ananya Zutshi, Vincent P. Willard, Charles A. Gersbach, and Farshid Guilak. “CRISPR/Cas9 Editing of Murine Induced Pluripotent Stem Cells for Engineering Inflammation-Resistant Tissues.” Arthritis & Rheumatology (Hoboken, N.J.) 69, no. 5 (May 2017): 1111–21. https://doi.org/10.1002/art.39982.Full Text
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Brunger, Jonathan M., Ananya Zutshi, Vincent P. Willard, Charles A. Gersbach, and Farshid Guilak. “Genome Engineering of Stem Cells for Autonomously Regulated, Closed-Loop Delivery of Biologic Drugs.” Stem Cell Reports 8, no. 5 (May 2017): 1202–13. https://doi.org/10.1016/j.stemcr.2017.03.022.Full Text
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Barrangou, Rodolphe, and Charles A. Gersbach. “Expanding the CRISPR Toolbox: Targeting RNA with Cas13b.” Molecular Cell 65, no. 4 (February 2017): 582–84. https://doi.org/10.1016/j.molcel.2017.02.002.Full Text
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Hilton, Isaac B., and Charles A. Gersbach. “Genetic engineering: Chemical control for CRISPR editing.” Nature Chemical Biology 13, no. 1 (January 2017): 2–3. https://doi.org/10.1038/nchembio.2243.Full Text
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Housden, Benjamin E., Matthias Muhar, Matthew Gemberling, Charles A. Gersbach, Didier Y. R. Stainier, Geraldine Seydoux, Stephanie E. Mohr, Johannes Zuber, and Norbert Perrimon. “Loss-of-function genetic tools for animal models: cross-species and cross-platform differences.” Nature Reviews. Genetics 18, no. 1 (January 2017): 24–40. https://doi.org/10.1038/nrg.2016.118.Full Text
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Lee, Jaewoo, Li Xu, Tyler M. Gibson, Charles A. Gersbach, and Bruce A. Sullenger. “Differential effects of toll-like receptor stimulation on mRNA-driven myogenic conversion of human and mouse fibroblasts.” Biochem Biophys Res Commun 478, no. 3 (September 23, 2016): 1484–90. https://doi.org/10.1016/j.bbrc.2016.08.159.Full Text Link to Item
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Robinson-Hamm, Jacqueline N., and Charles A. Gersbach. “Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.” Human Genetics 135, no. 9 (September 2016): 1029–40. https://doi.org/10.1007/s00439-016-1725-z.Full Text
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Black, Joshua B., Andrew F. Adler, Hong-Gang Wang, Anthony M. D’Ippolito, Hunter A. Hutchinson, Timothy E. Reddy, Geoffrey S. Pitt, Kam W. Leong, and Charles A. Gersbach. “Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells.” Cell Stem Cell 19, no. 3 (September 1, 2016): 406–14. https://doi.org/10.1016/j.stem.2016.07.001.Full Text Link to Item
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Moutos, Franklin T., Katherine A. Glass, Sarah A. Compton, Alison K. Ross, Charles A. Gersbach, Farshid Guilak, and Bradley T. Estes. “Anatomically shaped tissue-engineered cartilage with tunable and inducible anticytokine delivery for biological joint resurfacing.” Proceedings of the National Academy of Sciences of the United States of America 113, no. 31 (August 2016): E4513–22. https://doi.org/10.1073/pnas.1601639113.Full Text
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Nelson, Christopher E., and Charles A. Gersbach. “Engineering Delivery Vehicles for Genome Editing.” Annual Review of Chemical and Biomolecular Engineering 7 (June 2016): 637–62. https://doi.org/10.1146/annurev-chembioeng-080615-034711.Full Text
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Hwang, Priscilla Y., Liufang Jing, Jun Chen, Foon-Lian Lim, Ruhang Tang, Hyowon Choi, Kenneth M. Cheung, et al. “N-cadherin is Key to Expression of the Nucleus Pulposus Cell Phenotype under Selective Substrate Culture Conditions.” Scientific Reports 6 (June 2016): 28038. https://doi.org/10.1038/srep28038.Full Text
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Landau, Dustin J., Elizabeth Drake Brooks, Pablo Perez-Pinera, Hiruni Amarasekara, Adam Mefferd, Songtao Li, Andrew Bird, Charles A. Gersbach, and Dwight D. Koeberl. “In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.” Mol Ther 24, no. 4 (April 2016): 697–706. https://doi.org/10.1038/mt.2016.35.Full Text Link to Item
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Josephs, Eric A., D Dewran Kocak, Christopher J. Fitzgibbon, Joshua McMenemy, Charles A. Gersbach, and Piotr E. Marszalek. “Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage.” Nucleic Acids Research 44, no. 5 (March 2016): 2474. https://doi.org/10.1093/nar/gkv1293.Full Text
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Maeder, Morgan L., and Charles A. Gersbach. “Genome-editing Technologies for Gene and Cell Therapy.” Molecular Therapy : The Journal of the American Society of Gene Therapy 24, no. 3 (March 2016): 430–46. https://doi.org/10.1038/mt.2016.10.Full Text
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Nelson, Christopher E., and Charles A. Gersbach. “Cas9 loosens its grip on off-target sites.” Nature Biotechnology 34, no. 3 (March 2016): 298–99. https://doi.org/10.1038/nbt.3501.Full Text
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Thakore, Pratiksha I., Joshua B. Black, Isaac B. Hilton, and Charles A. Gersbach. “Editing the epigenome: technologies for programmable transcription and epigenetic modulation.” Nature Methods 13, no. 2 (February 2016): 127–37. https://doi.org/10.1038/nmeth.3733.Full Text
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Nelson, Christopher E., Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, Eirik A. Moreb, Ruth M. Castellanos Rivera, Sarina Madhavan, et al. “In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.” Science 351, no. 6271 (January 22, 2016): 403–7. https://doi.org/10.1126/science.aad5143.Full Text Link to Item
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Ousterout, David G., and Charles A. Gersbach. “The Development of TALE Nucleases for Biotechnology.” Methods in Molecular Biology (Clifton, N.J.) 1338 (January 2016): 27–42. https://doi.org/10.1007/978-1-4939-2932-0_3.Full Text
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Thakore, Pratiksha I., and Charles A. Gersbach. “Design, Assembly, and Characterization of TALE-Based Transcriptional Activators and Repressors.” Methods in Molecular Biology (Clifton, N.J.) 1338 (January 2016): 71–88. https://doi.org/10.1007/978-1-4939-2932-0_7.Full Text
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Doudna, Jennifer A., and Charles A. Gersbach. “Genome editing: the end of the beginning.” Genome Biology 16 (December 2015): 292. https://doi.org/10.1186/s13059-015-0860-5.Full Text
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Thakore, Pratiksha I., Anthony M. D’Ippolito, Lingyun Song, Alexias Safi, Nishkala K. Shivakumar, Ami M. Kabadi, Timothy E. Reddy, Gregory E. Crawford, and Charles A. Gersbach. “Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements.” Nat Methods 12, no. 12 (December 2015): 1143–49. https://doi.org/10.1038/nmeth.3630.Full Text Link to Item
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Hilton, Isaac B., and Charles A. Gersbach. “Enabling functional genomics with genome engineering.” Genome Research 25, no. 10 (October 2015): 1442–55. https://doi.org/10.1101/gr.190124.115.Full Text
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Josephs, Eric A., D Dewran Kocak, Christopher J. Fitzgibbon, Joshua McMenemy, Charles A. Gersbach, and Piotr E. Marszalek. “Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage.” Nucleic Acids Research 43, no. 18 (October 2015): 8924–41. https://doi.org/10.1093/nar/gkv892.Full Text
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Polstein, Lauren R., Pablo Perez-Pinera, D Dewran Kocak, Christopher M. Vockley, Peggy Bledsoe, Lingyun Song, Alexias Safi, Gregory E. Crawford, Timothy E. Reddy, and Charles A. Gersbach. “Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators.” Genome Res 25, no. 8 (August 2015): 1158–69. https://doi.org/10.1101/gr.179044.114.Full Text Link to Item
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Kabadi, Ami M., Pratiksha I. Thakore, Christopher M. Vockley, David G. Ousterout, Tyler M. Gibson, Farshid Guilak, Timothy E. Reddy, and Charles A. Gersbach. “Enhanced MyoD-induced transdifferentiation to a myogenic lineage by fusion to a potent transactivation domain.” Acs Synth Biol 4, no. 6 (June 19, 2015): 689–99. https://doi.org/10.1021/sb500322u.Full Text Link to Item
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Gibson, Tyler M., and Charles A. Gersbach. “Single-molecule analysis of myocyte differentiation reveals bimodal lineage commitment.” Integrative Biology : Quantitative Biosciences From Nano to Macro 7, no. 6 (June 2015): 663–71. https://doi.org/10.1039/c5ib00057b.Full Text
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Frank, Christopher L., Fang Liu, Ranjula Wijayatunge, Lingyun Song, Matthew T. Biegler, Marty G. Yang, Christopher M. Vockley, et al. “Regulation of chromatin accessibility and Zic binding at enhancers in the developing cerebellum.” Nat Neurosci 18, no. 5 (May 2015): 647–56. https://doi.org/10.1038/nn.3995.Full Text Link to Item
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Hilton, Isaac B., Anthony M. D’Ippolito, Christopher M. Vockley, Pratiksha I. Thakore, Gregory E. Crawford, Timothy E. Reddy, and Charles A. Gersbach. “Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers.” Nat Biotechnol 33, no. 5 (May 2015): 510–17. https://doi.org/10.1038/nbt.3199.Full Text Link to Item
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Diekman, Brian O., Pratiksha I. Thakore, Shannon K. O’Connor, Vincent P. Willard, Jonathan M. Brunger, Nicolas Christoforou, Kam W. Leong, Charles A. Gersbach, and Farshid Guilak. “Knockdown of the cell cycle inhibitor p21 enhances cartilage formation by induced pluripotent stem cells.” Tissue Engineering. Part A 21, no. 7–8 (April 2015): 1261–74. https://doi.org/10.1089/ten.tea.2014.0240.Full Text
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Ousterout, D. G., A. M. Kabadi, P. I. Thakore, P. Perez-Pinera, M. T. Brown, W. H. Majoros, T. E. Reddy, and C. A. Gersbach. “Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.” Molecular Therapy 23, no. 3 (March 5, 2015): 523–32. https://doi.org/10.1038/mt.2014.234.Full Text
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Ousterout, David G., Ami M. Kabadi, Pratiksha I. Thakore, Pablo Perez-Pinera, Matthew T. Brown, William H. Majoros, Timothy E. Reddy, and Charles A. Gersbach. “Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.” Mol Ther 23, no. 3 (March 2015): 523–32. https://doi.org/10.1038/mt.2014.234.Full Text Link to Item
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Polstein, Lauren R., and Charles A. Gersbach. “A light-inducible CRISPR-Cas9 system for control of endogenous gene activation.” Nature Chemical Biology 11, no. 3 (March 2015): 198–200. https://doi.org/10.1038/nchembio.1753.Full Text
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Ousterout, David G., Ami M. Kabadi, Pratiksha I. Thakore, William H. Majoros, Timothy E. Reddy, and Charles A. Gersbach. “Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.” Nat Commun 6 (February 18, 2015): 6244. https://doi.org/10.1038/ncomms7244.Full Text Link to Item
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Polstein, L. R., and C. A. Gersbach. “A light-inducible CRISPR-Cas9 system for control of endogenous gene activation.” Nature Chemical Biology 11, no. 3 (January 1, 2015): 198–200. https://doi.org/10.1038/nchembio.1753.Full Text
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Chakraborty, Syandan, HaYeun Ji, Ami M. Kabadi, Charles A. Gersbach, Nicolas Christoforou, and Kam W. Leong. “A CRISPR/Cas9-based system for reprogramming cell lineage specification.” Stem Cell Reports 3, no. 6 (December 2014): 940–47. https://doi.org/10.1016/j.stemcr.2014.09.013.Full Text
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Chakraborty, Syandan, HaYeun Ji, Jack Chen, Charles A. Gersbach, and Kam W. Leong. “Vector modifications to eliminate transposase expression following piggyBac-mediated transgenesis.” Scientific Reports 4 (December 2014): 7403. https://doi.org/10.1038/srep07403.Full Text
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Gersbach, Charles A. “Genome engineering: the next genomic revolution.” Nature Methods 11, no. 10 (October 2014): 1009–11. https://doi.org/10.1038/nmeth.3113.Full Text
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Kabadi, Ami M., and Charles A. Gersbach. “Special issue on engineered DNA-binding proteins.” Acs Synthetic Biology 3, no. 10 (October 2014): 702–3. https://doi.org/10.1021/sb500325e.Full Text
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Kabadi, Ami M., David G. Ousterout, Isaac B. Hilton, and Charles A. Gersbach. “Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.” Nucleic Acids Research 42, no. 19 (October 2014): e147. https://doi.org/10.1093/nar/gku749.Full Text
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Kabadi, Ami M., and Charles A. Gersbach. “Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression.” Methods (San Diego, Calif.) 69, no. 2 (September 2014): 188–97. https://doi.org/10.1016/j.ymeth.2014.06.014.Full Text
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Gersbach, Charles A., Thomas Gaj, and Carlos F. Barbas. “Synthetic zinc finger proteins: the advent of targeted gene regulation and genome modification technologies.” Accounts of Chemical Research 47, no. 8 (August 2014): 2309–18. https://doi.org/10.1021/ar500039w.Full Text
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Gersbach, Charles A., and Pablo Perez-Pinera. “Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine.” Expert Opinion on Therapeutic Targets 18, no. 8 (August 2014): 835–39. https://doi.org/10.1517/14728222.2014.913572.Full Text
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Glass, Katherine A., Jarrett M. Link, Jonathan M. Brunger, Franklin T. Moutos, Charles A. Gersbach, and Farshid Guilak. “Tissue-engineered cartilage with inducible and tunable immunomodulatory properties.” Biomaterials 35, no. 22 (July 2014): 5921–31. https://doi.org/10.1016/j.biomaterials.2014.03.073.Full Text
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High, Katherine, Philip D. Gregory, and Charles Gersbach. “CRISPR technology for gene therapy.” Nature Medicine 20, no. 5 (May 2014): 476–77. https://doi.org/10.1038/nm.3566.Full Text
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Brunger, Jonathan M., Nguyen P. T. Huynh, Caitlin M. Guenther, Pablo Perez-Pinera, Franklin T. Moutos, Johannah Sanchez-Adams, Charles A. Gersbach, and Farshid Guilak. “Scaffold-mediated lentiviral transduction for functional tissue engineering of cartilage.” Proceedings of the National Academy of Sciences of the United States of America 111, no. 9 (March 2014): E798–806. https://doi.org/10.1073/pnas.1321744111.Full Text
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Gersbach, C. A., T. Gaj, and C. F. Barbas. “Comparing genome editing technologies.” Genetic Engineering and Biotechnology News 34, no. 5 (March 1, 2014). https://doi.org/10.1089/gen.34.05.02.Full Text
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Polstein, Lauren R., and Charles A. Gersbach. “Light-inducible gene regulation with engineered zinc finger proteins.” Methods in Molecular Biology (Clifton, N.J.) 1148 (January 2014): 89–107. https://doi.org/10.1007/978-1-4939-0470-9_7.Full Text
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Ousterout, D. G., P. Perez-Pinera, P. I. Thakore, A. M. Kabadi, M. T. Brown, X. Qin, O. Fedrigo, V. Mouly, J. P. Tremblay, and C. A. Gersbach. “Erratum: Reading Frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients (Molecular Therapy (2013) 21 (1718-1726) DOI: 10.1038/mt.2013.111).” Molecular Therapy 21, no. 11 (November 1, 2013): 2130. https://doi.org/10.1038/mt.2013.229.Full Text
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Perez-Pinera, Pablo, D Dewran Kocak, Christopher M. Vockley, Andrew F. Adler, Ami M. Kabadi, Lauren R. Polstein, Pratiksha I. Thakore, et al. “RNA-guided gene activation by CRISPR-Cas9-based transcription factors.” Nat Methods 10, no. 10 (October 2013): 973–76. https://doi.org/10.1038/nmeth.2600.Full Text Link to Item
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Ousterout, David G., Pablo Perez-Pinera, Pratiksha I. Thakore, Ami M. Kabadi, Matthew T. Brown, Xiaoxia Qin, Olivier Fedrigo, Vincent Mouly, Jacques P. Tremblay, and Charles A. Gersbach. “Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.” Molecular Therapy : The Journal of the American Society of Gene Therapy 21, no. 9 (September 2013): 1718–26. https://doi.org/10.1038/mt.2013.111.Full Text
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Gersbach, C. A. “Engineered Proteins for Controlling Gene Expression” 1 (August 27, 2013): 125–38. https://doi.org/10.1016/B978-0-12-385942-6.00013-5.Full Text
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Gaj, Thomas, Charles A. Gersbach, and Carlos F. Barbas. “ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.” Trends in Biotechnology 31, no. 7 (July 2013): 397–405. https://doi.org/10.1016/j.tibtech.2013.04.004.Full Text
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Dumbauld, David W., Ted T. Lee, Ankur Singh, Jan Scrimgeour, Charles A. Gersbach, Evan A. Zamir, Jianping Fu, et al. “How vinculin regulates force transmission.” Proceedings of the National Academy of Sciences of the United States of America 110, no. 24 (June 2013): 9788–93. https://doi.org/10.1073/pnas.1216209110.Full Text
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Gibson, Tyler M., and Charles A. Gersbach. “The role of single-cell analyses in understanding cell lineage commitment.” Biotechnology Journal 8, no. 4 (April 2013): 397–407. https://doi.org/10.1002/biot.201200201.Full Text
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Bhakta, Mital S., Isabelle M. Henry, David G. Ousterout, Kumitaa Theva Das, Sarah H. Lockwood, Joshua F. Meckler, Mark C. Wallen, et al. “Highly active zinc-finger nucleases by extended modular assembly.” Genome Research 23, no. 3 (March 2013): 530–38. https://doi.org/10.1101/gr.143693.112.Full Text
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Perez-Pinera, Pablo, David G. Ousterout, Jonathan M. Brunger, Alicia M. Farin, Katherine A. Glass, Farshid Guilak, Gregory E. Crawford, Alexander J. Hartemink, and Charles A. Gersbach. “Synergistic and tunable human gene activation by combinations of synthetic transcription factors.” Nat Methods 10, no. 3 (March 2013): 239–42. https://doi.org/10.1038/nmeth.2361.Full Text Link to Item
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Tremblay, Jacques P., Xiao Xiao, Annemieke Aartsma-Rus, Carlos Barbas, Helen M. Blau, Adam J. Bogdanove, Kym Boycott, et al. “Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.” Molecular Therapy : The Journal of the American Society of Gene Therapy 21, no. 2 (February 2013): 266–68. https://doi.org/10.1038/mt.2013.4.Full Text
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Duvall, C. L., A. Prokop, C. A. Gersbach, and J. M. Davidson. “Gene Delivery into Cells and Tissues,” January 1, 2013, 687–723. https://doi.org/10.1016/B978-0-12-398358-9.00035-5.Full Text
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Gersbach, C. A., and C. F. Barbas. “Targeted plasmid integration into the Human Genome by engineered recombinases.” Topics in Current Genetics 23 (January 1, 2013): 267–84. https://doi.org/10.1007/978-94-007-4531-5_10.Full Text
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Ousterout, D. G., P. Perez-Pinera, P. I. Thakore, A. M. Kabadi, M. T. Brown, X. Qin, O. Fedrigo, V. Mouly, J. P. Tremblay, and C. A. Gersbach. “Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients.” Molecular Therapy 21, no. 9 (2013): 1718–26. https://doi.org/10.1038/mt.2013.111.Full Text
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Perez-Pinera, P., D. D. Kocak, C. M. Vockley, A. F. Adler, A. M. Kabadi, L. R. Polstein, P. I. Thakore, et al. “RNA-guided gene activation by CRISPR-Cas9-based transcription factors.” Nature Methods 10, no. 10 (2013): 973–76. https://doi.org/10.1038/nmeth.2600.Full Text
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Polstein, L. R., and C. A. Gersbach. “Photoregulated gene expression in human cells with light-inducible engineered transcription factors.” Asme 2012 Summer Bioengineering Conference, Sbc 2012, December 1, 2012, 351–52. https://doi.org/10.1115/SBC2012-80573.Full Text
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Polstein, Lauren R., and Charles A. Gersbach. “Light-inducible spatiotemporal control of gene activation by customizable zinc finger transcription factors.” Journal of the American Chemical Society 134, no. 40 (October 2012): 16480–83. https://doi.org/10.1021/ja3065667.Full Text
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Perez-Pinera, Pablo, David G. Ousterout, and Charles A. Gersbach. “Advances in targeted genome editing.” Current Opinion in Chemical Biology 16, no. 3–4 (August 2012): 268–77. https://doi.org/10.1016/j.cbpa.2012.06.007.Full Text
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Perez Pinera, P., D. G. Ousterout, M. T. Brown, and C. A. Gersbach. “Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases.” Nucleic Acids Research 40, no. 8 (2012): 3741–52.
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Gersbach, C. A. “Engineered Proteins for Controlling Gene Expression,” December 1, 2011, 159–76. https://doi.org/10.1016/B978-0-12-381422-7.10008-2.Full Text
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Gersbach, C. A. “Engineered bioactive molecules” 5 (October 1, 2011): 131–45.
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Gersbach, Charles A., Thomas Gaj, Russell M. Gordley, Andrew C. Mercer, and Carlos F. Barbas. “Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase.” Nucleic Acids Research 39, no. 17 (September 2011): 7868–78. https://doi.org/10.1093/nar/gkr421.Full Text
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Gaj, T., A. C. Mercer, C. A. Gersbach, R. M. Gordley, and C. F. Barbas. “Structure-guided reprogramming of serine recombinase DNA sequence specificity.” Proceedings of the National Academy of Sciences of the United States of America 108, no. 2 (January 11, 2011): 510–15. https://doi.org/10.1073/pnas.1016462108.Full Text
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Gaj, Thomas, Andrew C. Mercer, Charles A. Gersbach, Russell M. Gordley, and Carlos F. Barbas. “Structure-guided reprogramming of serine recombinase DNA sequence specificity.” Proceedings of the National Academy of Sciences of the United States of America 108, no. 2 (January 2011): 498–503. https://doi.org/10.1073/pnas.1014214108.Full Text
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Gersbach, Charles A., Thomas Gaj, Russell M. Gordley, and Carlos F. Barbas. “Directed evolution of recombinase specificity by split gene reassembly.” Nucleic Acids Research 38, no. 12 (July 2010): 4198–4206. https://doi.org/10.1093/nar/gkq125.Full Text
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Gordley, Russell M., Charles A. Gersbach, and Carlos F. Barbas. “Synthesis of programmable integrases.” Proceedings of the National Academy of Sciences of the United States of America 106, no. 13 (March 2009): 5053–58. https://doi.org/10.1073/pnas.0812502106.Full Text
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Gersbach, Charles A., Sean R. Coyer, Joseph M. Le Doux, and Andrés J. García. “Biomaterial-mediated retroviral gene transfer using self-assembled monolayers.” Biomaterials 28, no. 34 (December 2007): 5121–27. https://doi.org/10.1016/j.biomaterials.2007.07.047.Full Text
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Pregizer, Steven, Artem Barski, Charles A. Gersbach, Andrés J. García, and Baruch Frenkel. “Identification of novel Runx2 targets in osteoblasts: cell type-specific BMP-dependent regulation of Tram2.” Journal of Cellular Biochemistry 102, no. 6 (December 2007): 1458–71. https://doi.org/10.1002/jcb.21366.Full Text
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Gersbach, Charles A., Robert E. Guldberg, and Andrés J. García. “In vitro and in vivo osteoblastic differentiation of BMP-2- and Runx2-engineered skeletal myoblasts.” Journal of Cellular Biochemistry 100, no. 5 (April 2007): 1324–36. https://doi.org/10.1002/jcb.21118.Full Text
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Gersbach, Charles A., Jennifer E. Phillips, and Andrés J. García. “Genetic engineering for skeletal regenerative medicine.” Annual Review of Biomedical Engineering 9 (January 2007): 87–119. https://doi.org/10.1146/annurev.bioeng.9.060906.151949.Full Text
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Phillips, Jennifer E., Charles A. Gersbach, and Andrés J. García. “Virus-based gene therapy strategies for bone regeneration.” Biomaterials 28, no. 2 (January 2007): 211–29. https://doi.org/10.1016/j.biomaterials.2006.07.032.Full Text
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Gersbach, C. A., J. M. Le Doux, R. E. Guldberg, and A. J. García. “Inducible regulation of Runx2-stimulated osteogenesis.” Gene Therapy 13, no. 11 (June 2006): 873–82. https://doi.org/10.1038/sj.gt.3302725.Full Text
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Phillips, Jennifer E., Charles A. Gersbach, Abigail M. Wojtowicz, and Andrés J. García. “Glucocorticoid-induced osteogenesis is negatively regulated by Runx2/Cbfa1 serine phosphorylation.” Journal of Cell Science 119, no. Pt 3 (February 2006): 581–91. https://doi.org/10.1242/jcs.02758.Full Text
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Gersbach, C. A., J. E. Phillips, R. E. Guldberg, and A. J. García. “Runx2-genetically engineered cells for bone tissue engineering.” Proceedings of the 2005 Summer Bioengineering Conference 2005 (December 1, 2005): 603–4.
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Lan, Michael A., Charles A. Gersbach, Kristin E. Michael, Benjamin G. Keselowsky, and Andrés J. García. “Myoblast proliferation and differentiation on fibronectin-coated self assembled monolayers presenting different surface chemistries.” Biomaterials 26, no. 22 (August 2005): 4523–31. https://doi.org/10.1016/j.biomaterials.2004.11.028.Full Text
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Drevs, J., U. Zirrgiebel, C. I. M. Schmidt-Gersbach, K. Mross, M. Medinger, L. Lee, J. Pinheiro, et al. “Soluble markers for the assessment of biological activity with PTK787/ZK 222584 (PTK/ZK), a vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor in patients with advanced colorectal cancer from two phase I trials.” Annals of Oncology : Official Journal of the European Society for Medical Oncology 16, no. 4 (April 2005): 558–65. https://doi.org/10.1093/annonc/mdi118.Full Text
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Gersbach, C. A., B. A. Byers, R. E. Guldberg, G. K. Pavlath, and A. J. Garcia. “Runx2-stimulated transdifferentiation of primary skeletal myoblasts into an osteoblastic mineralizing phenotype for bone tissue engineering.” Transactions 7th World Biomaterials Congress, December 1, 2004, 328.
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Gersbach, Charles A., Benjamin A. Byers, Grace K. Pavlath, Robert E. Guldberg, and Andrés J. García. “Runx2/Cbfa1-genetically engineered skeletal myoblasts mineralize collagen scaffolds in vitro.” Biotechnology and Bioengineering 88, no. 3 (November 2004): 369–78. https://doi.org/10.1002/bit.20251.Full Text
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Gersbach, Charles A., Benjamin A. Byers, Grace K. Pavlath, and Andrés J. García. “Runx2/Cbfa1 stimulates transdifferentiation of primary skeletal myoblasts into a mineralizing osteoblastic phenotype.” Experimental Cell Research 300, no. 2 (November 2004): 406–17. https://doi.org/10.1016/j.yexcr.2004.07.031.Full Text
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GERSBACH, C. A. “Runx2/Cbfa1 stimulates transdifferentiation of primary skeletal myoblasts into a mineralizing osteoblastic phenotype.” Exp. Cell Res. 300 (2004): 406–17.
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García, Andrés J., Robert E. Guldberg, Benjamin A. Byers, Charles A. Gersbach, and Jennifer E. Phillips. “Addressing cell-sourcing limitations with gene therapy.” Ieee Engineering in Medicine and Biology Magazine : The Quarterly Magazine of the Engineering in Medicine & Biology Society 22, no. 5 (September 2003): 65–70. https://doi.org/10.1109/memb.2003.1256274.Full Text
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Book Sections
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Nelson, C. E., C. L. Duvall, A. Prokop, C. A. Gersbach, and J. M. Davidson. “Gene delivery into cells and tissues.” In Principles of Tissue Engineering, 519–54, 2020. https://doi.org/10.1016/B978-0-12-818422-6.00030-7.Full Text
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Nelson, C. E., and C. A. Gersbach. “Genome Editing for Duchenne Muscular Dystrophy.” In Muscle Gene Therapy, Second Edition, 383–403, 2019. https://doi.org/10.1007/978-3-030-03095-7_22.Full Text
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Ousterout, D. G., and C. A. Gersbach. “Genome Editing for Neuromuscular Diseases.” In Advances in Experimental Medicine and Biology, 895:51–79, 2016. https://doi.org/10.1007/978-1-4939-3509-3_4.Full Text
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Thakore, P. I., and C. A. Gersbach. “Genome Engineering for Therapeutic Applications.” In Translating Gene Therapy to the Clinic: Techniques and Approaches, 27–43, 2015. https://doi.org/10.1016/B978-0-12-800563-7.00003-8.Full Text
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Polstein, L. R., and C. A. Gersbach. “Spatiotemporal genetic control of cellular systems.” In Tissue and Organ Regeneration: Advances in Micro- and Nanotechnology, 156–97, 2014. https://doi.org/10.4032/9789814411684.Full Text
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Conference Papers
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Pickar-Oliver, Adrian, Joshua B. Black, Mae M. Lewis, Kevin J. Mutchnick, Tyler S. Klann, Kylie A. Gilcrest, Madeleine J. Sitton, et al. “Targeted transcriptional modulation with type I CRISPR-Cas systems in human cells.” In Nat Biotechnol, 37:1493–1501, 2019. https://doi.org/10.1038/s41587-019-0235-7.Full Text Link to Item
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Farhang, N., J. M. Brunger, J. D. Stover, P. I. Thakore, B. D. Lawrence, F. Guilak, C. A. Gersbach, L. A. Setton, and R. D. Bowles. “CRISPRi Immunomodulation for Tissue Engineering/Stem Cell Therapies Targeting Intervertebral Disc Degeneration.” In Tissue Engineering Part A, 21:S170–S170. MARY ANN LIEBERT, INC, 2015.Link to Item
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Glass, K. A., A. K. Ross, S. A. Compton, C. A. Gersbach, F. T. Moutos, B. T. Estes, and F. Guilak. “Anatomically-Shaped Tissue-Engineered Cartilage with Tunable and Inducible Anti-Inflammatory Capabilities.” In Tissue Engineering Part A, 21:S330–S330. MARY ANN LIEBERT, INC, 2015.Link to Item
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Black, Joshua, Andrew Adler, Hunter Hutchinson, Honggang Wang, Geoffrey Pitt, Kam Leong, and Charles Gersbach. “59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype.” In Molecular Therapy, 23:S26–S26. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33664-4.Full Text
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Schulz, Eric, Thorsten Bergmann, Maren Gebbing, Verena Schildgen, Oliver Schildgen, Charles Gersbach, and Anja Ehrhardt. “Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods.” In Molecular Therapy, 22:S126–27. NATURE PUBLISHING GROUP, 2014.Link to Item
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Perez-Pinera, Pablo, David G. Ousterout, Jonathan M. Brunger, Alicia M. Farin, Katherine A. Glass, Farshid Guilak, Gregory E. Crawford, Alexander J. Hartemink, and Charles A. Gersbach. “Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors.” In Molecular Therapy, 21:S93–S93. NATURE PUBLISHING GROUP, 2013.Link to Item
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Glass, K. A., J. M. Brunger, C. A. Gersbach, and F. Guilak. “Tunable expression of IL-1Ra in genetically modified mesenchymal stem cells for cartilage tissue engineering.” In Osteoarthritis and Cartilage, 21:S282–83. Elsevier BV, 2013. https://doi.org/10.1016/j.joca.2013.02.591.Full Text
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- Teaching & Mentoring
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Recent Courses
- BME 394: Projects in Biomedical Engineering (GE) 2023
- BME 564L: Genome Engineering Lab (GE, MC) 2023
- EGR 393: Research Projects in Engineering 2023
- BME 493: Projects in Biomedical Engineering (GE) 2022
- BME 562: Biology by Design (GE, MC) 2022
- BME 590L: Special Topics with Lab 2022
- EGR 393: Research Projects in Engineering 2022
- BME 493: Projects in Biomedical Engineering (GE) 2021
- BME 494: Projects in Biomedical Engineering (GE) 2021
- BME 562: Biology by Design (GE, MC) 2021
- BME 590L: Special Topics with Lab 2021
- BME 791: Graduate Independent Study 2021
- BME 899: Special Readings in Biomedical Engineering 2021
- CELLBIO 493: Research Independent Study 2021
- EGR 393: Research Projects in Engineering 2021
- Scholarly, Clinical, & Service Activities
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Outreach & Engaged Scholarship
- Bass Connections Faculty Team Leader. Duke Undergraduate International Genetically Engineered Machine. 2018 - 2019 2018 - 2019
- Bass Connections Faculty Team Member. Duke Undergraduate International Genetically Engineered Machine (iGEM) Team. August 2017 - May 2018 2017 - 2018
- Bass Connections Faculty Team Member. Duke Undergraduate International Genetically Engineered Machine (iGEM) Team. August 2016 - May 2017 2016 - 2017
- Bass Connections Faculty Team Member. International Genetically Engineered Machine (iGEM). May 2015 - May 2016 2015 - 2016
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