Aravind Asokan | Scholars@Duke

Aravind Asokan
Professor in Surgery

Synthetic Virology & Gene Therapy

Current Research Interests

Virology
RNA Biology
Viral Engineering
RNA Engineering
Gene Therapy
Gene Editing
Gene Regulation
Regenerative Medicine

Current Appointments & Affiliations

Professor in Surgery, Surgery, Surgical Sciences, Surgery 2019
Director of Gene Therapy, Surgery, Surgical Sciences, Surgery 2018
Professor of Molecular Genetics and Microbiology, Molecular Genetics and Microbiology, Basic Science Departments 2019
Professor of Biomedical Engineering, Biomedical Engineering, Pratt School of Engineering 2019
Affiliate of the Duke Regeneration Center, Regeneration Next Initiative, Basic Science Departments 2021

Contact Information

3 Genome Court, Msrb3, 5148, Box 103051, Duke Surgery, Durham, NC 27710
aravind.asokan@duke.edu
Asokan Lab for Synthetic Virology & Gene Therapy

Background
Education, Training, & Certifications
- Ph.D., University of North Carolina - Chapel Hill 2004
Previous Appointments & Affiliations
- Instructor in the Department of Surgery, Surgery, Surgical Sciences, Surgery 2018
Leadership & Clinical Positions at Duke
- Director of Gene Therapy
  Translating Duke Health Scholar
  Professor
  Department of Surgery
  Department of Molecular Genetics & Microbiology
  Department of Biomedical Engineering
Recognition
In the News
- DEC 13, 2022
  
  Gene Therapy for Heart Attacks in Mice Just Got More Precise
Research
Selected Grants
- University Training Program in Biomolecular and Tissue Engineering awarded by National Institutes of Health 1994 - 2027
- Genetic engineering of kidney allografts by ex vivo perfusion delivery of adeno-associated viral vectors awarded by National Institutes of Health 2022 - 2027
- Gene therapy for glycogen storage disease type III awarded by National Institutes of Health 2022 - 2027
- Dissecting AAV silencing in humanized mice awarded by National Institutes of Health 2023 - 2027
- Myovascular Mechanisms of Cardiac Growth and Regeneration awarded by National Institutes of Health 2022 - 2026
- Evaluating Wnt-Modulators as a Therapy for Treatment of Arrhythmic Cardiomyopathy In Vivo awarded by Hartwell Foundation 2022 - 2025
- Liver-directed AAV gene therapy for PHKG2-Glycogen Storage Disease IX (GSD IX y2) awarded by National Institutes of Health 2021 - 2024
- Targeted Therapy for Pompe Disease awarded by National Institutes of Health 2019 - 2024
- Evolving High Potency AAV Vectors for Neuromuscular Genome Editing awarded by National Institutes of Health 2018 - 2023
- LUMICKS C-TRAP awarded by National Institutes of Health 2022 - 2023
- Epigenome Editing Technologies for Treating Diverse Disease awarded by National Institutes of Health 2019 - 2023
- AAV-Exosomes: Escaping Neutralizing Antibody and Enhancing Delivery awarded by Icahn School of Medicine at Mount Sinai 2019 - 2023
- Determinants of AAV Tropism awarded by National Institutes of Health 2008 - 2023
- Evolving novel AAV vectors for gene therapy to cure HIV awarded by Oregon Health & Science University 2022 - 2023
- Next Generation AAV Vectors for Kidney Gene Therapy awarded by BridgeBio Gene Therapy Research, Inc. 2021 - 2023
- Reduction of Brain AVM Severity through Inhibition of Pathogenic Angiogenesis awarded by University of California - San Francisco 2020 - 2023
- AAV gene therapy for the treatment of Glycogen Storage Disease IX y2 awarded by Kriya Therapeutics 2022
- Editing the Kidney Epigenome awarded by University of Washington 2021 - 2022
- CircRNAs and CNS Gene Transfer awarded by National Institutes of Health 2018 - 2022
- RE-ENGINEERING AAV GENOME PACKAGING awarded by National Institutes of Health 2018 - 2022
- Characterization of Recombinant Adeno-Associated Viral Vectors awarded by StrideBio LLC 2019 - 2022
- CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular Dystrophy awarded by National Institutes of Health 2016 - 2021
- NONHUMAN PRIMATE TESTING CENTER FOR EVALUATION OF SOMATIC CELL GENOME EDITING TOOLS awarded by University of California - Davis 2019 - 2020
External Relationships
- AstraZeneca
- Atsena Therapeutics
- BridgeBio
- Isolere Bio, Inc
- Kriya Therapeutics, Inc.
- Mammoth Biosciences Inc
- Ring Therapeutics
- Sarepta Therapeutics
- StrideBio, Inc.
- TorqueBio LLC
Publications & Artistic Works
Selected Publications
- Academic Articles
  - Nyberg, William A., Jonathan Ark, Angela To, Sylvanie Clouden, Gabriella Reeder, Joseph J. Muldoon, Jing-Yi Chung, et al. “An evolved AAV variant enables efficient genetic engineering of murine T cells.” Cell 186, no. 2 (January 19, 2023): 446-460.e19. https://doi.org/10.1016/j.cell.2022.12.022.
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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.
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  - Konkimalla, Arvind, Satoshi Konishi, Yoshihiko Kobayashi, Preetish Kadur Lakshminarasimha Murthy, Lauren Macadlo, Ananya Mukherjee, Zachary Elmore, et al. “Multi-apical polarity of alveolar stem cells and their dynamics during lung development and regeneration.” Iscience 25, no. 10 (October 21, 2022): 105114. https://doi.org/10.1016/j.isci.2022.105114.
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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.
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  - Das, Anshuman, Madhuvanthi Vijayan, Eric M. Walton, V Grace Stafford, David N. Fiflis, and Aravind Asokan. “Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex.” J Virol 96, no. 4 (February 23, 2022): e0203921. https://doi.org/10.1128/JVI.02039-21.
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  - Emmanuel, Shanan N., J Kennon Smith, Jane Hsi, Yu-Shan Tseng, Matias Kaplan, Mario Mietzsch, Paul Chipman, Aravind Asokan, Robert McKenna, and Mavis Agbandje-McKenna. “Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants.” J Virol 96, no. 3 (February 9, 2022): e0125121. https://doi.org/10.1128/JVI.01251-21.
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  - Gao, Qimeng, Isabel F. DeLaura, Imran J. Anwar, Samuel J. Kesseli, Riley Kahan, Nader Abraham, Aravind Asokan, Andrew S. Barbas, and Matthew G. Hartwig. “Gene Therapy: Will the Promise of Optimizing Lung Allografts Become Reality?” Front Immunol 13 (2022): 931524. https://doi.org/10.3389/fimmu.2022.931524.
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  - Vekstein, Andrew M., David C. Wendell, Sophia DeLuca, Ruorong Yan, Yifan Chen, Muath Bishawi, Garth W. Devlin, et al. “Targeted Delivery for Cardiac Regeneration: Comparison of Intra-coronary Infusion and Intra-myocardial Injection in Porcine Hearts.” Front Cardiovasc Med 9 (2022): 833335. https://doi.org/10.3389/fcvm.2022.833335.
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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.
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  - Elmore, Zachary C., L. Patrick Havlik, Daniel K. Oh, Leif Anderson, George Daaboul, Garth W. Devlin, Heather A. Vincent, and Aravind Asokan. “The membrane associated accessory protein is an adeno-associated viral egress factor.” Nat Commun 12, no. 1 (October 29, 2021): 6239. https://doi.org/10.1038/s41467-021-26485-4.
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  - Havlik, L Patrick, Anshuman Das, Mario Mietzsch, Daniel K. Oh, Jonathan Ark, Robert McKenna, Mavis Agbandje-McKenna, and Aravind Asokan. “Receptor Switching in Newly Evolved Adeno-associated Viruses.” J Virol 95, no. 19 (September 9, 2021): e0058721. https://doi.org/10.1128/JVI.00587-21.
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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.
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  - Mitchell-Dick, Aaron, and Aravind Asokan. “AAV-CNS matters turn from gray to white.” Mol Ther 29, no. 5 (May 5, 2021): 1659–60. https://doi.org/10.1016/j.ymthe.2021.04.012.
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  - Hunanyan, Arsen S., Boris Kantor, Ram S. Puranam, Courtney Elliott, Angela McCall, Justin Dhindsa, Promila Pagadala, et al. “Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.” Hum Gene Ther 32, no. 7–8 (April 2021): 405–19. https://doi.org/10.1089/hum.2020.191.
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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.
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  - “A Beautiful Mind and the Heart of an Explorer.” Human Gene Therapy 32, no. 7–8 (April 1, 2021): 321–26. https://doi.org/10.1089/hum.2021.29158.mam.
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  - Meganck, Rita M., Jiacheng Liu, Andrew E. Hale, Katherine E. Simon, Marco M. Fanous, Heather A. Vincent, Jeremy E. Wilusz, Nathaniel J. Moorman, William F. Marzluff, and Aravind Asokan. “Engineering highly efficient backsplicing and translation of synthetic circRNAs.” Mol Ther Nucleic Acids 23 (March 5, 2021): 821–34. https://doi.org/10.1016/j.omtn.2021.01.003.
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  - Cannon, Paula, Aravind Asokan, Agnieszka Czechowicz, Paula Hammond, Donald B. Kohn, Andre Lieber, Punam Malik, et al. “Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development.” Hum Gene Ther 32, no. 1–2 (January 2021): 31–42. https://doi.org/10.1089/hum.2020.263.
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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.
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  - Kang, Martin H., Jiabiao Hu, Richard E. Pratt, Conrad P. Hodgkinson, Aravind Asokan, and Victor J. Dzau. “Optimizing delivery for efficient cardiac reprogramming.” Biochem Biophys Res Commun 533, no. 1 (November 26, 2020): 9–16. https://doi.org/10.1016/j.bbrc.2020.08.104.
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  - Madigan, Victoria J., Garrett E. Berry, Tyne O. Tyson, Dasean Nardone-White, Jonathan Ark, Zachary C. Elmore, Giridhar Murlidharan, Heather A. Vincent, and Aravind Asokan. “The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.” J Virol 94, no. 21 (October 14, 2020). https://doi.org/10.1128/JVI.01604-20.
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  - Elmore, Zachary C., Daniel K. Oh, Katherine E. Simon, Marco M. Fanous, and Aravind Asokan. “Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme.” Jci Insight 5, no. 19 (September 17, 2020). https://doi.org/10.1172/jci.insight.139881.
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  - Havlik, L Patrick, Katherine E. Simon, J Kennon Smith, Kelli A. Klinc, Longping V. Tse, Daniel K. Oh, Marco M. Fanous, et al. “Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach.” J Virol 94, no. 19 (September 15, 2020). https://doi.org/10.1128/JVI.00976-20.
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  - Das, Anshuman, Rodell Barrientos, Tomoyuki Shiota, Victoria Madigan, Ichiro Misumi, Kevin L. McKnight, Lu Sun, et al. “Gangliosides are essential endosomal receptors for quasi-enveloped and naked hepatitis A virus.” Nat Microbiol 5, no. 9 (September 2020): 1069–78. https://doi.org/10.1038/s41564-020-0727-8.
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  - Li, Jixue, Yanjie Li, Jun Wang, Trevor J. Gonzalez, Aravind Asokan, Jill S. Napierala, and Marek Napierala. “Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.” Hum Gene Ther 31, no. 15–16 (August 2020): 839–51. https://doi.org/10.1089/hum.2020.053.
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  - Huang, Qiang, Malkiel A. Cohen, Fernando C. Alsina, Garth Devlin, Aliesha Garrett, Jennifer McKey, Patrick Havlik, et al. “Intravital imaging of mouse embryos.” Science 368, no. 6487 (April 10, 2020): 181–86. https://doi.org/10.1126/science.aba0210.
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  - Madigan, Victoria J., Tyne O. Tyson, Julianne A. Yuziuk, Minakshi Pillai, Sven Moller-Tank, and Aravind Asokan. “A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes.” J Virol 93, no. 21 (November 1, 2019). https://doi.org/10.1128/JVI.00943-19.
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  - Abdulhaqq, Shaheed A., Helen Wu, John B. Schell, Katherine B. Hammond, Jason S. Reed, Alfred W. Legasse, Michael K. Axthelm, et al. “Vaccine-Mediated Inhibition of the Transporter Associated with Antigen Processing Is Insufficient To Induce Major Histocompatibility Complex E-Restricted CD8+ T Cells in Nonhuman Primates.” J Virol 93, no. 19 (October 1, 2019). https://doi.org/10.1128/JVI.00592-19.
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  - Asokan, Aravind. “CRISPR genome editing in stem cells turns to gold.” Nat Mater 18, no. 10 (October 2019): 1038–39. https://doi.org/10.1038/s41563-019-0491-4.
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  - Madigan, Victoria J., Julianne A. Yuziuk, Anna M. Chiarella, Tyne O. Tyson, Rita M. Meganck, Zachary C. Elmore, Longping V. Tse, Nathaniel A. Hathaway, and Aravind Asokan. “Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription.” Plos Pathog 15, no. 8 (August 2019): e1007988. https://doi.org/10.1371/journal.ppat.1007988.
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  - Albright, Blake H., Katherine E. Simon, Minakshi Pillai, Garth W. Devlin, and Aravind Asokan. “Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses.” J Virol 93, no. 11 (June 1, 2019). https://doi.org/10.1128/JVI.00332-19.
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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.
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  - Mestre, Humberto, Lauren M. Hablitz, Anna Lr Xavier, Weixi Feng, Wenyan Zou, Tinglin Pu, Hiromu Monai, et al. “Aquaporin-4-dependent glymphatic solute transport in the rodent brain.” Elife 7 (December 18, 2018). https://doi.org/10.7554/eLife.40070.
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  - Meganck, Rita M., Erin K. Borchardt, Ruth M. Castellanos Rivera, Miranda L. Scalabrino, Jeremy E. Wilusz, William F. Marzluff, and Aravind Asokan. “Tissue-Dependent Expression and Translation of Circular RNAs with Recombinant AAV Vectors In Vivo.” Mol Ther Nucleic Acids 13 (December 7, 2018): 89–98. https://doi.org/10.1016/j.omtn.2018.08.008.
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  - Castle, Michael J., Yuhsiang Cheng, Aravind Asokan, and Mark H. Tuszynski. “Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion.” Sci Adv 4, no. 11 (November 2018): eaau9859. https://doi.org/10.1126/sciadv.aau9859.
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  - Tse, Longping V., Sven Moller-Tank, Rita M. Meganck, and Aravind Asokan. “Mapping and Engineering Functional Domains of the Assembly-Activating Protein of Adeno-associated Viruses.” J Virol 92, no. 14 (July 15, 2018). https://doi.org/10.1128/JVI.00393-18.
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  - Crowther, Andrew J., Szu-Aun Lim, Brent Asrican, Blake H. Albright, Josh Wooten, Chia-Yu Yeh, Hechen Bao, et al. “An Adeno-Associated Virus-Based Toolkit for Preferential Targeting and Manipulating Quiescent Neural Stem Cells in the Adult Hippocampus.” Stem Cell Reports 10, no. 3 (March 13, 2018): 1146–59. https://doi.org/10.1016/j.stemcr.2018.01.018.
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  - Albright, Blake H., Claire M. Storey, Giridhar Murlidharan, Ruth M. Castellanos Rivera, Garrett E. Berry, Victoria J. Madigan, and Aravind Asokan. “Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier.” Mol Ther 26, no. 2 (February 7, 2018): 510–23. https://doi.org/10.1016/j.ymthe.2017.10.017.
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  - Burwitz, Benjamin J., Jochen M. Wettengel, Martin A. Mück-Häusl, Marc Ringelhan, Chunkyu Ko, Marvin M. Festag, Katherine B. Hammond, et al. “Hepatocytic expression of human sodium-taurocholate cotransporting polypeptide enables hepatitis B virus infection of macaques.” Nat Commun 8, no. 1 (December 15, 2017): 2146. https://doi.org/10.1038/s41467-017-01953-y.
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  - Guo, Jiami, James M. Otis, Holden Higginbotham, Chase Monckton, JrGang Cheng, Aravind Asokan, Kirk Mykytyn, Tamara Caspary, Garret D. Stuber, and E. S. Anton. “Primary Cilia Signaling Shapes the Development of Interneuronal Connectivity.” Dev Cell 42, no. 3 (August 7, 2017): 286-300.e4. https://doi.org/10.1016/j.devcel.2017.07.010.
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  - Tse, Longping Victor, Kelli A. Klinc, Victoria J. Madigan, Ruth M. Castellanos Rivera, Lindsey F. Wells, L Patrick Havlik, J Kennon Smith, Mavis Agbandje-McKenna, and Aravind Asokan. “Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion.” Proc Natl Acad Sci U S A 114, no. 24 (June 13, 2017): E4812–21. https://doi.org/10.1073/pnas.1704766114.
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  - Borchardt, Erin K., Rita M. Meganck, Heather A. Vincent, Christopher B. Ball, Silvia B. V. Ramos, Nathaniel J. Moorman, William F. Marzluff, and Aravind Asokan. “Inducing circular RNA formation using the CRISPR endoribonuclease Csy4.” Rna 23, no. 5 (May 2017): 619–27. https://doi.org/10.1261/rna.056838.116.
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  - Tarantal, Alice F., C Chang I. Lee, Michele L. Martinez, Aravind Asokan, and R Jude Samulski. “Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.” Hum Gene Ther 28, no. 5 (May 2017): 385–91. https://doi.org/10.1089/hum.2016.130.
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  - Zeng, Cheng, Sven Moller-Tank, Aravind Asokan, and Bogdan Dragnea. “Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2.” J Phys Chem B 121, no. 8 (March 2, 2017): 1843–53. https://doi.org/10.1021/acs.jpcb.6b10131.
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  - Berry, Garrett Edward, and Aravind Asokan. “Cellular transduction mechanisms of adeno-associated viral vectors.” Curr Opin Virol 21 (December 2016): 54–60. https://doi.org/10.1016/j.coviro.2016.08.001.
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  - Tseng, Yu-Shan, Kim Van Vliet, Lavanya Rao, Robert McKenna, Barry J. Byrne, Aravind Asokan, and Mavis Agbandje-McKenna. “Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies.” J Virol Methods 236 (October 2016): 105–10. https://doi.org/10.1016/j.jviromet.2016.07.009.
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  - Murlidharan, Giridhar, Andrew Crowther, Rebecca A. Reardon, Juan Song, and Aravind Asokan. “Glymphatic fluid transport controls paravascular clearance of AAV vectors from the brain.” Jci Insight 1, no. 14 (September 8, 2016): e88034. https://doi.org/10.1172/jci.insight.88034.
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  - Murlidharan, Giridhar, Kensuke Sakamoto, Lavanya Rao, Travis Corriher, Dan Wang, Guangping Gao, Patrick Sullivan, and Aravind Asokan. “CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector.” Mol Ther Nucleic Acids 5, no. 7 (July 19, 2016): e338. https://doi.org/10.1038/mtna.2016.49.
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  - Pierson, Elizabeth E., David Z. Keifer, Aravind Asokan, and Martin F. Jarrold. “Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry.” Anal Chem 88, no. 13 (July 5, 2016): 6718–25. https://doi.org/10.1021/acs.analchem.6b00883.
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  - Huang, Lin-Ya, Ami Patel, Robert Ng, Edward Blake Miller, Sujata Halder, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site.” J Virol 90, no. 11 (June 1, 2016): 5219–30. https://doi.org/10.1128/JVI.00161-16.
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  - Madigan, Victoria J., and Aravind Asokan. “Engineering AAV receptor footprints for gene therapy.” Curr Opin Virol 18 (June 2016): 89–96. https://doi.org/10.1016/j.coviro.2016.05.001.
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  - Leeuw, Charles N. de, Andrea J. Korecki, Garrett E. Berry, Jack W. Hickmott, Siu Ling Lam, Tess C. Lengyell, Russell J. Bonaguro, et al. “rAAV-compatible MiniPromoters for restricted expression in the brain and eye.” Mol Brain 9, no. 1 (May 10, 2016): 52. https://doi.org/10.1186/s13041-016-0232-4.
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  - Vance, Melisa, Telmo Llanga, Will Bennett, Kenton Woodard, Giridhar Murlidharan, Neil Chungfat, Aravind Asokan, et al. “AAV Gene Therapy for MPS1-associated Corneal Blindness.” Sci Rep 6 (February 22, 2016): 22131. https://doi.org/10.1038/srep22131.
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  - Li, Chengwen, Shuqing Wu, Blake Albright, Matthew Hirsch, Wuping Li, Yu-Shan Tseng, Mavis Agbandje-McKenna, Scott McPhee, Aravind Asokan, and R Jude Samulski. “Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.” Mol Ther 24, no. 1 (February 2016): 53–65. https://doi.org/10.1038/mt.2015.134.
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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.
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  - Berry, Garrett E., and Aravind Asokan. “Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction.” J Biol Chem 291, no. 2 (January 8, 2016): 939–47. https://doi.org/10.1074/jbc.M115.687657.
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  - Borchardt, Erin K., Leonidas A. Vandoros, Michael Huang, Patrick E. Lackey, William F. Marzluff, and Aravind Asokan. “Controlling mRNA stability and translation with the CRISPR endoribonuclease Csy4.” Rna 21, no. 11 (November 2015): 1921–30. https://doi.org/10.1261/rna.051227.115.
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  - Schreiber, Claire A., Toshie Sakuma, Yoshihiro Izumiya, Sara J. Holditch, Raymond D. Hickey, Robert K. Bressin, Upamanyu Basu, Kazunori Koide, Aravind Asokan, and Yasuhiro Ikeda. “An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses.” Plos Pathog 11, no. 8 (August 2015): e1005082. https://doi.org/10.1371/journal.ppat.1005082.
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  - Tse, Longping V., Sven Moller-Tank, and Aravind Asokan. “Strategies to circumvent humoral immunity to adeno-associated viral vectors.” Expert Opin Biol Ther 15, no. 6 (June 2015): 845–55. https://doi.org/10.1517/14712598.2015.1035645.
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  - Hemphill, James, Erin K. Borchardt, Kalyn Brown, Aravind Asokan, and Alexander Deiters. “Optical Control of CRISPR/Cas9 Gene Editing.” J Am Chem Soc 137, no. 17 (May 6, 2015): 5642–45. https://doi.org/10.1021/ja512664v.
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  - Murlidharan, Giridhar, Travis Corriher, H Troy Ghashghaei, and Aravind Asokan. “Unique glycan signatures regulate adeno-associated virus tropism in the developing brain.” J Virol 89, no. 7 (April 2015): 3976–87. https://doi.org/10.1128/JVI.02951-14.
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  - Shen, Shen, Garrett E. Berry, Ruth M. Castellanos Rivera, Roland Y. Cheung, Andrew N. Troupes, Sarah M. Brown, Tal Kafri, and Aravind Asokan. “Functional analysis of the putative integrin recognition motif on adeno-associated virus 9.” J Biol Chem 290, no. 3 (January 16, 2015): 1496–1504. https://doi.org/10.1074/jbc.M114.608281.
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  - Ishikawa, Kiyotake, Kenneth M. Fish, Lisa Tilemann, Kleopatra Rapti, Jaume Aguero, Carlos G. Santos-Gallego, Ahyoung Lee, et al. “Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.” Mol Ther 22, no. 12 (December 2014): 2038–45. https://doi.org/10.1038/mt.2014.127.
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  - Pleticha, Josef, Lukas F. Heilmann, Christopher H. Evans, Aravind Asokan, Richard Jude Samulski, and Andreas S. Beutler. “Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs.” Mol Pain 10 (September 2, 2014): 54. https://doi.org/10.1186/1744-8069-10-54.
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  - O’Reilly, Marina, Howard J. Federoff, Yuman Fong, Donald B. Kohn, Amy P. Patterson, Nabil Ahmed, Aravind Asokan, et al. “Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.” Hum Gene Ther 25, no. 6 (June 2014): 488–97. https://doi.org/10.1089/hum.2014.045.
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  - Murlidharan, Giridhar, Richard J. Samulski, and Aravind Asokan. “Biology of adeno-associated viral vectors in the central nervous system.” Front Mol Neurosci 7 (2014): 76. https://doi.org/10.3389/fnmol.2014.00076.
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  - Shen, Shen, Andrew N. Troupes, Nagesh Pulicherla, and Aravind Asokan. “Multiple roles for sialylated glycans in determining the cardiopulmonary tropism of adeno-associated virus 4.” J Virol 87, no. 24 (December 2013): 13206–13. https://doi.org/10.1128/JVI.02109-13.
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  - Asokan, Aravind, and R Jude Samulski. “An emerging adeno-associated viral vector pipeline for cardiac gene therapy.” Hum Gene Ther 24, no. 11 (November 2013): 906–13. https://doi.org/10.1089/hum.2013.2515.
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  - Shen, Shen, Eric D. Horowitz, Andrew N. Troupes, Sarah M. Brown, Nagesh Pulicherla, Richard J. Samulski, Mavis Agbandje-McKenna, and Aravind Asokan. “Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency.” J Biol Chem 288, no. 40 (October 4, 2013): 28814–23. https://doi.org/10.1074/jbc.M113.482380.
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  - Horowitz, Eric D., K Shefaet Rahman, Brian D. Bower, David J. Dismuke, Michael R. Falvo, Jack D. Griffith, Stephen C. Harvey, and Aravind Asokan. “Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release.” J Virol 87, no. 6 (March 2013): 2994–3002. https://doi.org/10.1128/JVI.03017-12.
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  - Shen, Shen, Kelli D. Bryant, Junjiang Sun, Sarah M. Brown, Andrew Troupes, Nagesh Pulicherla, and Aravind Asokan. “Glycan binding avidity determines the systemic fate of adeno-associated virus type 9.” J Virol 86, no. 19 (October 2012): 10408–17. https://doi.org/10.1128/JVI.01155-12.
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  - Li, Chengwen, Nina Diprimio, Dawn E. Bowles, Matthew L. Hirsch, Paul E. Monahan, Aravind Asokan, Joseph Rabinowitz, Mavis Agbandje-McKenna, and R Jude Samulski. “Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles.” J Virol 86, no. 15 (August 2012): 7752–59. https://doi.org/10.1128/JVI.00675-12.
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  - Horowitz, Eric D., M. G. Finn, and Aravind Asokan. “Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection.” Acs Chem Biol 7, no. 6 (June 15, 2012): 1059–66. https://doi.org/10.1021/cb3000265.
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  - Denard, Jerome, Cyriaque Beley, Robert Kotin, René Lai-Kuen, Stéphane Blot, Hervé Leh, Aravind Asokan, et al. “Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.” J Virol 86, no. 12 (June 2012): 6620–31. https://doi.org/10.1128/JVI.00297-12.
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  - Asokan, Aravind, David V. Schaffer, and R Jude Samulski. “The AAV vector toolkit: poised at the clinical crossroads.” Mol Ther 20, no. 4 (April 2012): 699–708. https://doi.org/10.1038/mt.2011.287.
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  - Li, C., N. Narkbunnam, R. J. Samulski, A. Asokan, G. Hu, L. J. Jacobson, M. J. Manco-Johnson, P. E. Monahan, and P. E. Joint Outcome Study Investigators. “Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.” Gene Ther 19, no. 3 (March 2012): 288–94. https://doi.org/10.1038/gt.2011.90.
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  - Pulicherla, Nagesh, Pradeep Kota, Nikolay V. Dokholyan, and Aravind Asokan. “Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsids.” Plos One 7, no. 2 (2012): e32163. https://doi.org/10.1371/journal.pone.0032163.
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  - Gray, Steven J., Vivian W. Choi, Aravind Asokan, Rebecca A. Haberman, Thomas J. McCown, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration.” Curr Protoc Neurosci Chapter 4 (October 2011): Unit-4.17. https://doi.org/10.1002/0471142301.ns0417s57.
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  - Pulicherla, N., and A. Asokan. “Peptide affinity reagents for AAV capsid recognition and purification.” Gene Ther 18, no. 10 (October 2011): 1020–24. https://doi.org/10.1038/gt.2011.46.
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  - Pulicherla, Nagesh, Shen Shen, Swati Yadav, Kari Debbink, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and Aravind Asokan. “Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.” Mol Ther 19, no. 6 (June 2011): 1070–78. https://doi.org/10.1038/mt.2011.22.
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  - Horowitz, Eric D., Marc S. Weinberg, and Aravind Asokan. “Glycated AAV vectors: chemical redirection of viral tissue tropism.” Bioconjug Chem 22, no. 4 (April 20, 2011): 529–32. https://doi.org/10.1021/bc100477g.
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  - Shen, Shen, Kelli D. Bryant, Sarah M. Brown, Scott H. Randell, and Aravind Asokan. “Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.” J Biol Chem 286, no. 15 (April 15, 2011): 13532–40. https://doi.org/10.1074/jbc.M110.210922.
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  - Kapoor, Amit, Mady Hornig, Aravind Asokan, Brent Williams, Jose A. Henriquez, and W Ian Lipkin. “Bocavirus episome in infected human tissue contains non-identical termini.” Plos One 6, no. 6 (2011): e21362. https://doi.org/10.1371/journal.pone.0021362.
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  - Phillips, Jana L., Julia Hegge, Jon A. Wolff, R Jude Samulski, and Aravind Asokan. “Systemic gene transfer to skeletal muscle using reengineered AAV vectors.” Methods Mol Biol 709 (2011): 141–51. https://doi.org/10.1007/978-1-61737-982-6_9.
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  - Asokan, Aravind. “Reengineered AAV vectors: old dog, new tricks.” Discov Med 9, no. 48 (May 2010): 399–403.
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  - Asokan, Aravind, Julia C. Conway, Jana L. Phillips, Chengwen Li, Julia Hegge, Rebecca Sinnott, Swati Yadav, et al. “Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle.” Nat Biotechnol 28, no. 1 (January 2010): 79–82. https://doi.org/10.1038/nbt.1599.
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  - Li, Chengwen, Matt Hirsch, Nina DiPrimio, Aravind Asokan, Kevin Goudy, Roland Tisch, and R Jude Samulski. “Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo.” J Virol 83, no. 13 (July 2009): 6817–24. https://doi.org/10.1128/JVI.00278-09.
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  - Li, Chengwen, Kevin Goudy, Matt Hirsch, Aravind Asokan, Yun Fan, Jeff Alexander, Junjiang Sun, et al. “Cellular immune response to cryptic epitopes during therapeutic gene transfer.” Proc Natl Acad Sci U S A 106, no. 26 (June 30, 2009): 10770–74. https://doi.org/10.1073/pnas.0902269106.
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  - Li, C., M. Hirsch, P. Carter, A. Asokan, X. Zhou, Z. Wu, and R. J. Samulski. “A small regulatory element from chromosome 19 enhances liver-specific gene expression.” Gene Ther 16, no. 1 (January 2009): 43–51. https://doi.org/10.1038/gt.2008.134.
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  - Asokan, A., J. S. Johnson, C. Li, and R. J. Samulski. “Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals.” Gene Ther 15, no. 24 (December 2008): 1618–22. https://doi.org/10.1038/gt.2008.127.
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  - Li, Wuping, Aravind Asokan, Zhijian Wu, Terry Van Dyke, Nina DiPrimio, Jarrod S. Johnson, Lakshmanan Govindaswamy, et al. “Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.” Mol Ther 16, no. 7 (July 2008): 1252–60. https://doi.org/10.1038/mt.2008.100.
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  - DiPrimio, Nina, Aravind Asokan, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and R Jude Samulski. “Surface loop dynamics in adeno-associated virus capsid assembly.” J Virol 82, no. 11 (June 2008): 5178–89. https://doi.org/10.1128/JVI.02721-07.
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  - Li, Chengwen, Matthew Hirsch, Aravind Asokan, Brian Zeithaml, Hong Ma, Tal Kafri, and R Jude Samulski. “Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo.” J Virol 81, no. 14 (July 2007): 7540–47. https://doi.org/10.1128/JVI.00529-07.
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  - Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors for in vitro and in vivo use.” Curr Protoc Mol Biol Chapter 16 (April 2007): Unit-16.25. https://doi.org/10.1002/0471142727.mb1625s78.
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  - Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors.” Curr Protoc Hum Genet Chapter 12 (April 2007): Unit-12.9. https://doi.org/10.1002/0471142905.hg1209s53.
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  - Wu, Zhijian, Aravind Asokan, Joshua C. Grieger, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and R Jude Samulski. “Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes.” J Virol 80, no. 22 (November 2006): 11393–97. https://doi.org/10.1128/JVI.01288-06.
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  - Asokan, Aravind, Julie B. Hamra, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and Richard J. Samulski. “Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry.” J Virol 80, no. 18 (September 2006): 8961–69. https://doi.org/10.1128/JVI.00843-06.
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  - Wu, Zhijian, Aravind Asokan, and R Jude Samulski. “Adeno-associated virus serotypes: vector toolkit for human gene therapy.” Mol Ther 14, no. 3 (September 2006): 316–27. https://doi.org/10.1016/j.ymthe.2006.05.009.
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  - Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, Thomas J. McCown, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administration.” Curr Protoc Neurosci Chapter 4 (May 2006): Unit-4.17. https://doi.org/10.1002/0471142301.ns0417s35.
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  - Asokan, Aravind, and R Jude Samulski. “AAV does the shuffle.” Nat Biotechnol 24, no. 2 (February 2006): 158–60. https://doi.org/10.1038/nbt0206-158.
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  - Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules 4. Head group-dependent membrane permeabilization by pH-sensitive detergents.” J Control Release 106, no. 1–2 (August 18, 2005): 146–53. https://doi.org/10.1016/j.jconrel.2005.04.010.
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  - Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules. II. Mechanistic studies with pH-sensitive morpholine lipids.” Biochim Biophys Acta 1611, no. 1–2 (April 1, 2003): 151–60. https://doi.org/10.1016/s0005-2736(03)00050-6.
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  - Chen, Feng-Jing, Aravind Asokan, and Moo J. Cho. “Cytosolic delivery of macromolecules: I. Synthesis and characterization of pH-sensitive acyloxyalkylimidazoles.” Biochim Biophys Acta 1611, no. 1–2 (April 1, 2003): 140–50. https://doi.org/10.1016/s0005-2736(03)00049-x.
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  - Asokan, Aravind, and Moo J. Cho. “Exploitation of intracellular pH gradients in the cellular delivery of macromolecules.” J Pharm Sci 91, no. 4 (April 2002): 903–13. https://doi.org/10.1002/jps.10095.
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- Book Sections
  - Murlidharan, G., R. J. Samulski, and A. Asokan. “Gene therapy of CNS disorders using recombinant AAV vectors.” In Translational Neuroscience: Fundamental Approaches for Neurological Disorders, 9–24, 2016. https://doi.org/10.1007/978-1-4899-7654-3_2.
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  - Asokan, A., R. Cheung, and M. J. Cho. “Strategies for the Cytosolic Delivery of Macromolecules: An Overview.” In Handbook of Pharmaceutical Biotechnology, 279–96, 2006. https://doi.org/10.1002/9780470117118.ch3c.
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- Conference Papers
  - Ark, Jonathan I., William Nyberg, Katherine Simon, Alan Rosales, Sylvanie Clouden, Justin Eyquem, and Aravind Asokan. “Targeted In Vivo Manipulation of Murine T-Cells Using a Newly Evolved AAV Capsid Mutant.” In Molecular Therapy, 30:425–26, 2022.
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  - Ark, Jonathan I., William Nyberg, Sylvanie Clouden, Patrick L. Havlik, Justin Eyquem, and Aravind Asokan. “Evolution of a New AAV Variant with Murine T Lymphocyte Tropism Using the MHC-Ib Molecule H2-Q7 as a Receptor.” In Molecular Therapy, 30:389–389, 2022.
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  - Austin, Bridget, Travis Gulledge, Cheryl Bandoski-Gralinski, Aravind Asokan, and Ruth Castellanos Rivera. “Pre-Existing Humoral Immunity to AAV Capsids: Species and Age-Related Differences in Anti-Capsid ELISA and Neutralizing Antibody Assays.” In Molecular Therapy, 30:340–340, 2022.
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  - Barzi, Mercedes, Collin Johnson, Tong Chen, Madeline Hemmingsen, Areeg El-Gharbawy, Trevor J. Gonzalez, Alan Rosales, et al. “Novel Gene Supplementation, Genome Editing and Cellular Therapeutic Approaches to Treat Glutaric Aciduria Type I.” In Molecular Therapy, 30:458–458, 2022.
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  - Chen, Tong, Mercedes Barzi, Francis Pankowicz, Trevor J. Gonzalez, Beatrice Bissig-Choisat, Aravind Asokan, and Karl-Dimiter Bissig. “A Novel Human Liver Chimeric Mouse Model Lacking the Murine AAVR Gene for Validation of AAV Gene Therapy Vectors.” In Molecular Therapy, 30:43–43, 2022.
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  - Elmore, Zac, Daniel Oh, and Aravind Asokan. “AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress.” In Molecular Therapy, 30:71–71, 2022.
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  - Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Deeksha Bali, Sarah Young, Rebecca Koch, Baodong Sun, Aravind Asokan, and Priya S. Kishnani. “SEVERE PROGRESSION OF LIVER DISEASE IN AN AGING PHKG2(-/-) MOUSE MODEL RECAPITULATES GSD IX G2 PATIENT PHENOTYPE.” In Molecular Genetics and Metabolism, 135:272–272, 2022.
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  - Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Rebecca L. Koch, Deeksha Bali, Sarah Young, William Jeck, Baodong Sun, Aravind Asokan, and Priya S. Kishnani. “Liver Directed AAV Gene Therapy Reverses Progression of Glycogen Storage Disease Type IX gamma 2 in Mice.” In Molecular Therapy, 30:478–79, 2022.
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  - Gonzalez, Trevor J., Leo Blondel, Alan Rosales, Heather Daniels, Charles A. Gersbach, and Aravind Asokan. “Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant.” In Molecular Therapy, 30:211–12, 2022.
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  - Gulledge, Travis, Bridget Austin, Cheryl Bandoski-Gralinski, Aravind Asokan, and Ruth Castellanos Rivera. “STRV5, a Liver Detargeted AAV Capsid Demonstrates Improved Safety Profile After Intravenous Administration of High Doses in Non-Human Primates.” In Molecular Therapy, 30:339–339, 2022.
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  - Hull, Joshua A., Mario Mietzsch, Yong Z. Tan, Sriram Aiyer, Antonette Bennett, Renuk Lakshman, Paul Chipman, et al. “AAV Capsid Dynamics at the 5-Fold Pore Controls Genome Release.” In Molecular Therapy, 30:205–6, 2022.
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  - Jeon, Kilsoo, Stephanie Gold, Mei Hu, J Kennon Smith, Colin O’Banion, Marc Morra, Tim S. Luongo, Aravind Asokan, and John D. Graef. “A Human iPSC-Derived Neuron and Cardiomyocyte Platform for Assessing Novel Recombinant Adeno-Associated Viral (AAV) Vectors.” In Molecular Therapy, 30:482–482, 2022.
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  - Kesseli, S. J., M. K. Krischak, Q. Gao, S. E. Halpern, M. Zhang, M. Song, T. J. Gonzalez, A. Asokan, A. S. Barbas, and M. G. Hartwig. “Intra-Tracheal Adeno-Associated Virus Mediates Gene Transduction During Static Cold Storage in Rodent Lung Transplantation.” In Journal of Heart and Lung Transplantation, 41:S38–39, 2022.
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  - Kesseli, S. J., M. K. Krischak, Q. Gao, T. Gonzalez, M. Zhang, S. E. Halpern, R. Kahan, et al. “Adeno-associated virus mediates gene transduction after static cold storage treatment in rodent lung transplantation.” In Journal of Thoracic and Cardiovascular Surgery, 2022. https://doi.org/10.1016/j.jtcvs.2022.08.050.
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  - Kesseli, Samuel, Madison Krischak, Qimeng Gao, Samantha Halpern, Min Zhang, Nader Abraham, Riley Kahan, et al. “Adeno-associated virus 9 mediates gene transduction during static cold storage in rodent liver transplantation.” In American Journal of Transplantation, 22:67–67, 2022.
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  - Loeb, Ezra J., L Patrick Havlik, and Aravind Asokan. “Unlocking Avian AAV Transduction in Mammalian Cells and Tissues for Immune Evasion and Redosing.” In Molecular Therapy, 30:204–204, 2022.
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  - Rosales, Alan, Trevor Gonzalez, and Aravind Asokan. “A Newly Evolved AAV Variant Enables Potent Gene Transfer in Kidneys of Multiple Species.” In Molecular Therapy, 30:555–555, 2022.
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  - Smith, Timothy J., Zachary C. Elmore, and Aravind Asokan. “Furin is a Host Factor Restricting Adeno-Associated Virus 4 Transduction.” In Molecular Therapy, 30:206–206, 2022.
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  - Walton, Eric M., Joshua B. Black, Laura Castillo, Charles A. Gersbach, and Aravind Asokan. “Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome.” In Molecular Therapy, 30:430–430, 2022.
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  - Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya S. Kishnani. “Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2^-/- mouse model.” In Molecular Genetics and Metabolism, 133:269–76, 2021. https://doi.org/10.1016/j.ymgme.2021.05.008.
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  - Ark, Jonathan, William Nyberg, Patrick Havlik, Angela To, Justin Eyquem, and Aravind Asokan. “Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations.” In Molecular Therapy, 29:32–32, 2021.
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  - Das, Anshuman, Patrick Havlik, Jonathan Ark, Mario Mietzsch, Mavis Agbandje-McKenna, and Aravind Asokan. “A CRISPR/Cas9 Genome Wide Screen and Cryo-EM Structure Reveal Receptor Switching by a Synthetic AAV Variant.” In Molecular Therapy, 29:151–52, 2021.
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  - Gibson, Rebecca, Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya Kishnani. “Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model.” In Molecular Therapy, 29:239–239, 2021.
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  - Havlik, Lawrence Patrick, Daniel K. Oh, Anshuman Das, Ruth M Castellanos Rivera, and Aravind Asokan. “Mechanistic Elucidation of Adenovirus Mediated Enhancement of Recombinant Adeno-Associated Virus (rAAV) towards Efficient Low Dose Gene Therapy.” In Molecular Therapy, 29:153–54, 2021.
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  - Havlik, Lawrence Patrick, Daniel K. Oh, Anshuman Das, Ruth M Castellanos Rivera, and Aravind Asokan. “Distinct Functional Roles for AAV Capsid Surface Epitopes Revealed through Structure-Guided Evolution.” In Molecular Therapy, 29:153–153, 2021.
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  - Meganck, Rita M., Sven Moller-Tank, Jiacheng Liu, Longping V. Tse, Heather A. Vincent, William F. Marzluff, and Aravind Asokan. “Subgenomic Flaviviral RNA Elements Enhance rAAV Vector mRNA Stability and Transduction.” In Molecular Therapy, 29:151–151, 2021.
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  - Vekstein, Andrew M., David C. Wendell, Dawn E. Bowles, Sophia DeLuca, Ruorong Yan, Muath Bishawi, Garth Devlin, et al. “Targeted Intra-Coronary Delivery versus Intra-Myocardial Injection of Therapeutics for Myocardial Recovery: A Nanoparticle Image Guided Porcine Study.” In Circulation, Vol. 144, 2021.
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  - Vijayan, Madhuvanthi, Anshuman Das, Grace Stafford, Zachary Elmore, Eric Walton, and Aravind Asokan. “The Human Silencing Hub (HUSH Complex) Is a Potent Regulator of AAV Transgene Silencing.” In Molecular Therapy, 29:47–47, 2021.
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  - Walton, Eric M., Joshua B. Black, Charles A. Gersbach, and Aravind Asokan. “A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes.” In Molecular Therapy, 29:150–51, 2021.
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  - Elmore, Zachary C., L Patrick Havlik, Daniel Oh, and Aravind Asokan. “The Membrane-Associated Accessory Protein (MAAP) is Essential for Rapid Extracellular Secretion of Adeno-Associated Viruses.” In Molecular Therapy, 28:44–44, 2020.
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  - Elmore, Zachary, Daniel Oh, Katherine Simon, Marco Fanous, and Aravind Asokan. “A Novel Approach for Rapid and Transient Depletion of Pre-Existing Neutralizing Antibodies Against AAV Vectors.” In Molecular Therapy, 28:398–398, 2020.
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  - Gonzalez, Trevor J., L Patrick Havlik, Marco Fanous, Katherine Simon, Laura Edwards, Jennifer Kwon, V Grace Stafford, et al. “Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation.” In Molecular Therapy, 28:12–13, 2020.
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  - Havlik, L Patrick, Zachary C. Elmore, Daniel K. Oh, Timothy J. Smith, and Aravind Asokan. “Evolving New AAV Strains that Demonstrate AAVR Independence.” In Molecular Therapy, 28:245–46, 2020.
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  - Kwon, Jennifer, Ashish Vankara, Joel Bohning, Garth Delvin, Aravind Asokan, and Charles Gersbach. “Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy.” In Molecular Therapy, 28:330–330, 2020.
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  - McCall, Angela L., Augusto de Leon, Anna F. Fusco, Aidan Bailey, Marco Fanous, Aravind Asokan, and Mai K. ElMallah. “A Novel Capsid for Smooth Muscle Pathology in Pompe Disease.” In Molecular Therapy, 28:492–492, 2020.
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  - Meganck, Rita M., Trevor J. Gonzalez, Heather A. Vincent, and Aravind Asokan. “Engineering Backsplicing Introns for Robust Expression of Synthetic Circular RNAs from AAV Vectors.” In Molecular Therapy, 28:21–21, 2020.
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  - Bulaklak, Karen, Jacqueline Robinson-Hamm, Veronica Gough, Christopher E. Nelson, Victoria Madigan, Aravind Asokan, and Charles A. Gersbach. “AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy.” In Molecular Therapy, 27:377–377. CELL PRESS, 2019.
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  - Castle, Michael J., Yuhsiang Cheng, Aravind Asokan, and Mark H. Tuszynski. “Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9.” In Molecular Therapy, 27:97–98. CELL PRESS, 2019.
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  - Gonzalez, Trevor J., Rita Meganck, Marco Fanous, Kate Simon, and Aravind Asokan. “Design Principles for AAV Mediated Circular RNA Expression in the Brain.” In Molecular Therapy, 27:60–60. CELL PRESS, 2019.
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  - Havlik, L Patrick, L Victor Tse, J Kennon Smith, Kelli A. Klinc, Daniel Oh, Katherine Simon, Wenwei Shao, Chengwen Li, Mavis Agbandje-McKenna, and Aravind Asokan. “Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution.” In Molecular Therapy, 27:53–53. CELL PRESS, 2019.
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  - Madigan, Victoria, Tyne O. Tyson, Julianne A. Yuziuk, Minakshi Pillai, Sven Moller-Tank, and Aravind Asokan. “A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor.” In Molecular Therapy, 27:424–25. CELL PRESS, 2019.
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  - Madigan, Victoria, Tyne O. Tyson, and Aravind Asokan. “Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription.” In Molecular Therapy, 27:425–425. CELL PRESS, 2019.
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  - Nelson, Christopher, Yaoying Wu, Matthew Gemberling, Matthew Oliver, Joel D. Bohning, Jacqueline N. Robinson-Hamm, Karen Bulaklak, et al. “Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy.” In Molecular Therapy, 27:46–47. CELL PRESS, 2019.
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  - Burwitz, Benjamin, Jochen Wettengel, Martin Muck-Hausl, Katherine Hammond, Marc Ringelhan, Chunkyu Ko, Jason Reed, et al. “A new HIV/HBV co-infection model: hepatocytic expression of human sodium taurocholate cotransporting polypeptide (NTCP) Enables Hepatitis B Virus Infection of Macaques.” In Journal of Medical Primatology, 47:324–324. WILEY, 2018.
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  - Albright, Blake H., Claire Storey, Garth Devlin, Mavis Agbandje-McKenna, and Aravind Asokan. “Capsid-Glycan Receptor Interactions Influence AAV Transport across the Blood-Brain Barrier.” In Molecular Therapy, 26:29–29. CELL PRESS, 2018.
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  - Madigan, Victoria J., Julianne Yuziuk, Longping V. Tse, Anna Chiarella, Tyne Tyson, Nate Hathaway, and Aravind Asokan. “RNF121 is a Key Transcriptional Regulator of AAV Genome Expression.” In Molecular Therapy, 26:27–27. CELL PRESS, 2018.
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  - Meganck, Rita M., Erin K. Bochardt, Ruth M. Castellanos, Miranda L. Scalabrino, Jeremy Wilusz, William F. Marzluff, and Aravind Asokan. “Systemic Expression of Translatable Circular Rnas Using Recombinant AAV Vectors.” In Molecular Therapy, 26:273–273. CELL PRESS, 2018.
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  - Nelson, Christopher, Matthew Gemberling, Matthew L. Oliver, Chady H. Hakim, Ruth M Castellanos Rivera, Aravind Asokan, Dongsheng Duan, and Charles A. Gersbach. “Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy.” In Molecular Therapy, 26:431–32. CELL PRESS, 2018.
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  - Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Matthew Gemberling, Veronica Gough, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9.” In Molecular Therapy, 26:118–19. CELL PRESS, 2018.
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  - Tse, Longping V., Sven Moller-Tank, Rita M. Meganck, Mavis Agbandje-McKenna, and Aravind Asokan. “Mapping and Engineering Functional Domains of the Assembly Activating Protein of Adeno-Associated Viruses.” In Molecular Therapy, 26:27–28. CELL PRESS, 2018.
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  - Albright, Blake, Claire Storey, Giridhar Murlidharan, Ruth Castellanos Rivera, and Aravind Asokan. “Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain Barrier.” In Molecular Therapy, 25:230–31. CELL PRESS, 2017.
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  - Moller-Tank, Sven, Ruth M. Castellanos, Daniel Oh, Longping V. Tse, Miranda L. Scalabrino, and Aravind Asokan. “Expanding the CNS Transduction Profile of AAV4 by Structure-Guided Evolution.” In Molecular Therapy, 25:247–247. CELL PRESS, 2017.
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  - Nelson, Christopher, Matthew Gemberling, Sarina Madhavan, Ruth M Castellanos Rivera, Aravind Asokan, and Charles A. Gersbach. “Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy.” In Molecular Therapy, 25:100–101. CELL PRESS, 2017.
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  - Rivera, Ruth M Castellanos, Austin D. Sun, Marco Fanous, and Aravind Asokan. “Disruption of Water Homeostasis Affects AAV Transport Across the Blood-Brain Barrier.” In Molecular Therapy, 25:109–109. CELL PRESS, 2017.
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  - Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Matthew Gemberling, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9.” In Molecular Therapy, 25:17–17. CELL PRESS, 2017.
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  - Smith, J Kennon S., Yu-Shan Tseng, Daniel Noe, Mathias Kaplan, Lavanya Rao, Robert McKenna, Sergei Zolotukhin, et al. “Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape Variants.” In Molecular Therapy, 25:337–337. CELL PRESS, 2017.
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  - Tse, Longping V., Kelli A. Klinc, Victoria J. Madigan, Ruth M Castellanos Rivera, Lindsey F. Wells, L Patrick Havlik, J Kennon Smith, Mavis Agbandje-McKenna, and Aravind Asokan. “Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene Transfer.” In Molecular Therapy, 25:232–232. CELL PRESS, 2017.
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  - Murlidharan, Giridhar, and Aravind Asokan. “Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer.” In Molecular Therapy, 24:S24–S24. NATURE PUBLISHING GROUP, 2016.
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  - Berry, Garrett, Giridhar Murlidharan, and Aravind Asokan. “30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS.” In Molecular Therapy, 24:S14–S14. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32839-8.
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  - Huang, Lin-Ya, Ami Patel, Robert Ng, Edward B. Miller, Sujata Halder, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “727. Characterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopes.” In Molecular Therapy, 24:S287–S287. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33535-3.
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  - Nelson, Christopher, Matthew Gemberling, Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, Ruth M. Castellanos, Sarina Madhavan, et al. “482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy.” In Molecular Therapy, 24:S191–S191. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33291-9.
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  - Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy.” In Molecular Therapy, 24:S201–S201. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33313-5.
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  - Schreiber, Claire A., Yoshihiro Izumiya, Aravind Asokan, and Yasuhiro Ikeda. “8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction.” In Molecular Therapy, 24:S4–5. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32817-9.
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  - Schreiber, C. A., T. Sakuma, Y. Izumiya, R. K. Bressin, U. Basu, K. Koide, A. Asokan, and Y. Ikeda. “Enhanced AAV vector transduction through genetic and pharmacological inhibition of U2 snRNP components.” In Human Gene Therapy, 26:A3–4. MARY ANN LIEBERT, INC, 2015.
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  - Woodard, Kenton, Katharine J. Liang, Thomas Lentz, William Bennett, Aravind Asokan, and Richard Samulski. “Transduction of Mouse Retina by Intravitreal Delivery Using Chimeric Capsids with Altered Glycan Interactions.” In Investigative Ophthalmology & Visual Science, Vol. 56. ASSOC RESEARCH VISION OPHTHALMOLOGY INC, 2015.
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  - Rivera, Ruth M Castellanos, Lavanya Rao, Giridhar Murlidharan, Travis Corriher, and Aravind Asokan. “Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors.” In Molecular Therapy, 23:S77–S77. NATURE PUBLISHING GROUP, 2015.
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  - Albright, Blake H., Victoria Madigan, Lavanya Rao, John A. Chiorini, Mavis Agbandje-McKenna, and Aravind Asokan. “302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors.” In Molecular Therapy, 23:S122–S122. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33911-9.
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  - Berry, Garrett E., and Aravind Asokan. “38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction.” In Molecular Therapy, 23:S17–S17. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33643-7.
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  - Murlidharan, Giridhar, Lavanya Rao, Dan Wang, Travis Corriher, Kyung Seok-Oh, Guangping Gao, R. Jude Samulski, Alice F. Tarantal, and Aravind Asokan. “14. Next Generation AAV Vectors for Limiting Systemic Leakage and Improving Safety Following CNS Administration.” In Molecular Therapy, 23:S6–S6. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33618-8.
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  - Murlidharan, Giridhar, and Aravind Asokan. “265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain.” In Molecular Therapy, 23:S106–S106. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33874-6.
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  - Nelson, Christopher E., Ruth M. Castellanos, Fei Ann Ran, Winston Yan, Feng Zhang, Aravind Asokan, and Charles A. Gersbach. “397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy.” In Molecular Therapy, 23:S157–58. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)34006-0.
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  - Rao, Lavanya, Blake H. Albright, Travis Corriher, Giridhar Murlidharan, and Aravind Asokan. “42. Differential Transduction Profiles of AAV Vectors in a Mouse Model of Human Glycosylation.” In Molecular Therapy, 23:S18–19. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33647-4.
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  - Schreiber, Claire, Toshie Sakuma, Yoshihiro Izumiya, Sara Holditch, Raymond Hickey, Robert Bressin, Upamanyu Basu, Kazunori Koide, Aravind Asokan, and Yasuhiro Ikeda. “41. U2 snRNP Spliceosome Proteins Block Recombinant AAV Vector Transduction.” In Molecular Therapy, 23:S18–S18. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33646-2.
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  - Huang, Lin-Ya, Robert Ng, Edward B. Miller, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “Characterizing Essential Receptor Binding Residues for AAV1.” In Molecular Therapy, 22:S2–S2. NATURE PUBLISHING GROUP, 2014.
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  - Simpson, Elizabeth M., Charles N. de Leeuw, Siu Ling Lam, Andrea J. Korecki, Russell J. Bonaguro, Kaelan Wong, Michelle Zhou, et al. “Regional-CNS MiniPromoters for AAV Are Identified in a High Through-Put Pipeline.” In Molecular Therapy, 22:S112–S112. NATURE PUBLISHING GROUP, 2014.
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  - Tseng, Yu-Shan, Kim Van Vliet, Matias Kaplan, J Kennon Smith, Lavanya Rao, Robert McKenna, Barry J. Byrne, Sergei Zolotukhin, Aravind Asokan, and Mavis Agbandje-McKenna. “Mapping the Antigenic Structure of Adeno-Associated Virus Serotypes 8 and 9.” In Molecular Therapy, 22:S1–S1. NATURE PUBLISHING GROUP, 2014.
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  - Murlidharan, Giridhar, Laura Sommerville, Troy Ghashghaei, and Aravind Asokan. “Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4.” In Molecular Therapy, 21:S1–S1. NATURE PUBLISHING GROUP, 2013.
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  - Shen, Shen, Andrew Troupes, Nagesh Pulicherla, and Aravind Asokan. “Sialic Acids and Erythrocyte Interactions Are Critical Determinants of Cardiopulmonary Tropism Displayed by AAV Serotype 4.” In Molecular Therapy, 21:S31–S31. NATURE PUBLISHING GROUP, 2013.
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  - Shen, Shen, and Aravind Asokan. “Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo.” In Molecular Therapy, 21:S30–S30. NATURE PUBLISHING GROUP, 2013.
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  - Denard, Jerome, Cyriaque Beley, Robert Kotin, Rene Lai-Kuen, Stephane Blot, Herve Leh, Aravind Asokan, et al. “Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.” In Human Gene Therapy, 23:A122–A122. MARY ANN LIEBERT INC, 2012.
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  - Horowitz, Eric D., and Aravind Asokan. “Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating.” In Molecular Therapy, 20:S202–S202. NATURE PUBLISHING GROUP, 2012.
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  - Shen, Shen, Junjiang Sun, Matthew Hirsch, Sai Chavala, Paul Monahan, and Aravind Asokan. “Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors.” In Molecular Therapy, 20:S203–S203. NATURE PUBLISHING GROUP, 2012.
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  - Monahan, P. E., C. Li, N. Narkbunnam, R. J. Samulski, G. Hu, A. Asokan, and M. J. Manco-Johnson. “Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.” In Journal of Thrombosis and Haemostasis, 9:231–32. WILEY-BLACKWELL, 2011.
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  - Xiao, Ping-Jie, Jarrod S. Johnson, Aaron Neumann, Xinghua Zeng, Aravind Asokan, and Richard J. Samulski. “Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells.” In Molecular Therapy, 17:S38–S38. NATURE PUBLISHING GROUP, 2009.
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  - Asokan, Aravind, Jarrod S. Johnson, and Richard J. Samulski. “739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue.” In Molecular Therapy, 13:S286–S286. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.821.
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  - Asokan, Aravind, Julie B. Hamra, Mavis Agbandje-McKenna, and Richard J. Samulski. “7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry.” In Molecular Therapy, 13:S3–S3. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.017.
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  - DiPrimio, Nina, Aravind Asokan, and Richard J. Samulski. “104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle.” In Molecular Therapy, 13:S43–S43. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.124.
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  - Asokan, Arvind, and Richard J. Samulski. “From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop.” In Mol Ther, 11:656–60, 2005. https://doi.org/10.1016/j.ymthe.2005.03.014.
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  - Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules. 3. Synthesis and characterization of acid-sensitive bis-detergents.” In Bioconjug Chem, 15:1166–73, 2004. https://doi.org/10.1021/bc049880n.
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