Charles Gersbach

John W. Strohbehn Distinguished Professor of Biomedical Engineering

Biomedical Engineering

Charles.gersbach@duke.edu

Box 90281, 136 Hudson Hall, Durham, NC 27708

2123 CIEMAS, Durham, NC 27708

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1. Education

Academic Experience
1. Current Duke Appointments & Affiliations
2. Previous Duke Appointments & Affiliations

Global Scholarship

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Research

Scholarly Works

Teaching & Mentoring
1. Recent Courses

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1. Academic Honors
2. News Items

Professional Activities
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Scholarly Works - Conferences

Discovery of dexamethasone-inducible transgene regulatory elements (TREs) via high-throughput in vivo screening with AAV vectors

Conference MOLECULAR THERAPY · May 8, 2026 Link to item Cite

Whole genome methylation analysis in exhausted T cells reveals targets for CRISPR epigenetic editors

Conference MOLECULAR THERAPY · May 8, 2026 Link to item Cite

A high-throughput and modular screening platform to optimize CAR designs for oncology and autoimmunity

Conference MOLECULAR THERAPY · May 8, 2026 Link to item Cite

Combinatorial screening of regulatory elements distinguishes transgene expression between cardiac and skeletal muscle in vivo

Conference MOLECULAR THERAPY · May 8, 2026 Link to item Cite

Abstract B021: CRISPRi screening reveals an enhancer-PLXNA1 axis as a critical regulator in ccRCC

Conference Cancer Research · March 13, 2026 AbstractBackground/Objective:Enhancers are critical cis-regulatory elements that modulate gene expression, and their dysregulat ... Full text Cite

Discovery and Characterization of Novel Epigenome Editing Domains

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Transcription Factor-Wide Screens to Identify Regulators of Tissue Resident Memory T cell States

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

In Vivo High-Throughput Screening of Regulatory Elements to Distinguish AAV Transgene Expression Between Cardiac and Skeletal Muscle

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Understanding Schizophrenia-Associated Loci Using iPSC-Derived Neurons and CRISPR Screening

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Epigenetic Reprogramming of Tumor-Infiltrating Lymphocytes for Improved AntiTumor Killing

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Engineering Functionally Mature iPSC-derived Hepatocytes through Mapping Transcription Factor Regulatory Mechanisms

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

A gene regulatory element modulates myosin expression and alters cellular stress response

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Direct Reprogramming of Primary Human Astrocytes to Functional Neurons with CRISPR-Based Epigenome Editing

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Epigenetic CRISPR Editors Encoded as mRNA for Engineering Primary Human T Cells

Conference MOLECULAR THERAPY · April 28, 2025 Link to item Cite

Respiratory pathology in the mdx/utrn-/- mouse: A murine model for Duchenne Muscular Dystrophy (DMD)

Conference Plos One · February 1, 2025 Duchenne muscular dystrophy (DMD) is an X-linked devastating disease caused by a lack of dystrophin which results in progressive muscle weakness. As muscle weakness progresses, respiratory insufficiency and hypoventilation result in significant morbidity a ... Full text Cite

T-Cell Epigenetic Reprogramming with TGIF2LX-Overexpression Enhances Adoptive Cell Therapy

Conference MOLECULAR THERAPY · April 22, 2024 Link to item Cite

Transient Delivery of Epigenome Editors Stably Represses PCSK9 and Lowers LDL Cholesterol in Non-Human Primates

Conference MOLECULAR THERAPY · May 12, 2023 Link to item Cite

AAV Serotype Tropism and Editing in Young Rhesus Monkeys

Conference MOLECULAR THERAPY · May 1, 2023 Link to item Cite

Modeling Gene Editing Outcomes in Microphysiological Human Tissue System Models of Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · May 1, 2023 Link to item Cite

CRISPR Genome Editing in Microphysiological Human Tissue System Models of Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

CRISPR-Based Epigenome Editing Screens in Primary Human T Cells

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

A Novel Cas9 System with Robust Genome and Epigenome Editing in Human Cells

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

A High-Throughput Screen for CRISPR-Cas9-Mediated Exon Deletion

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant

Conference MOLECULAR THERAPY · April 2022 Link to item Cite

Respiratory Pathology in a Humanized Mouse Model of Duchenne Muscular Dystrophy

Conference The FASEB Journal · May 2021 Duchenne muscular dystrophy (DMD) is the most common X‐linked disease affecting 1 in 3500 male births. DMD is characterized by mutations in the DMD gene, which ... Full text Cite

A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes

Conference MOLECULAR THERAPY · April 27, 2021 Link to item Cite

Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration

Conference MOLECULAR THERAPY · April 27, 2021 Link to item Cite

Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation

Conference MOLECULAR THERAPY · April 28, 2020 Link to item Cite

Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · April 28, 2020 Link to item Cite

Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration

Conference MOLECULAR THERAPY · April 28, 2020 Link to item Cite

Targeted Genomic Integration to Restore the NF1 Coding Sequence in Models of Neurofibromatosis Type I (NF1)

Conference MOLECULAR THERAPY · April 28, 2020 Link to item Cite

CRISPR-Mediated Gene Correction in a Severe Humanized Mouse Model of Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · April 28, 2020 Link to item Cite

Respiratory Dysfunction in a novel mouse model of Duchenne Muscular Dystrophy

Conference FASEB JOURNAL · April 2020 Full text Link to item Cite

Targeted transcriptional modulation with type I CRISPR-Cas systems in human cells.

Conference Nat Biotechnol · December 2019 Class 2 CRISPR-Cas systems, such as Cas9 and Cas12, have been widely used to target DNA sequences in eukaryotic genomes. However, class 1 CRISPR-Cas systems, which represent about 90% of all CRISPR systems in nature, remain largely unexplored for genome en ... Full text Link to item Cite

PCSK9 Knockdown Decreases Inflammatory Markers and U937 Monocyte Accumulation in a Tissue-Engineered Blood Vessel Model

Conference CIRCULATION · November 19, 2019 Link to item Cite

Directing Skeletal Myogenic Progenitor Cell Lineage Specification with CRISPR/Cas9-Based Transcriptional Activators

Conference MOLECULAR THERAPY · April 22, 2019 Link to item Cite

Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · April 22, 2019 Link to item Cite

Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration

Conference MOLECULAR THERAPY · April 22, 2019 Link to item Cite

AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · April 22, 2019 Link to item Cite

NEW THERAPEUTIC APPROACHES AND THEIR READOUT

Conference Neuromuscular Disorders · October 2018 Full text Cite

Genome editing as a therapy for Duchenne muscular dystrophy

Conference ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY · August 19, 2018 Link to item Cite

Optimization of the gRNA Vector Dose Enhances Long-Term Systemic AAV CRISPR Therapy in Muscular Dystrophy

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

Targeting Muscle Satellite Cells with Adeno-Associated Viral Vectors

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

Highly Efficient Chondrogenic Differentiation of Human iPSCs and Purification via a Reporter Allele Generated by CRISPR-Cas9 Genome Editing

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

AAV-9 CRISPR Mediated Satellite Cell Editing Restored Dystrophin Expression after Complete Degeneration in a Whole Muscle Graft Model

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9

Conference MOLECULAR THERAPY · May 1, 2018 Link to item Cite

Abstract A17: Generation and comparison of CRISPR/Cas9 and Cre-mediated genetically engineered mouse models of sarcoma

Conference Clinical Cancer Research · January 15, 2018 AbstractGenetically engineered mouse models (GEMMs) that employ site-specific recombinase (SSR) technology are important tools for cancer research, and recently the CRISPR/Cas9 system has been increasingly u ... Full text Cite

Abstract 2810: Using CRISPR/Cas9 to generate primary soft tissue sarcoma in genetically engineered and wild-type mice

Conference Cancer Research · July 1, 2017 AbstractGenetically engineered mouse models (GEMMs) that employ site-specific recombinase (SSR) technology are important tools for pre-clinical studies, but this approach is costly and time-consuming. Here, ... Full text Cite

In Vivo Gene Silencing with S-aureus CRISPR-Cas9 Repressors

Conference MOLECULAR THERAPY · May 1, 2017 Link to item Cite

Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy

Conference MOLECULAR THERAPY · May 1, 2017 Link to item Cite

Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9

Conference MOLECULAR THERAPY · May 1, 2017 Link to item Cite

Genome editing for Duchenne muscular dystrophy

Conference HUMAN GENE THERAPY · November 1, 2016 Link to item Cite

502. Biomaterial-Mediated Lentiviral Delivery of Anti-Inflammatory Genes in Cartilage-Derived Matrix Hemispheres

Conference Molecular Therapy · May 2016 Full text Cite

318. Targeted Genome Editing of Human Induced Pluripotent Stem Cells Using CRISPR/CAS9 to Generate a Knock-in Type II Collagen Reporter for the Purification of Chondrogenic Cells

Conference Molecular Therapy · May 2016 Full text Cite

311. In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Enhances Biochemical Correction in Mice with Glycogen Storage Disease Type IA

Conference Molecular Therapy · May 2016 Full text Cite

525. Directed Molecular Evolution of Transcription Factors in Mammalian Cells for Enhanced Directed Cell Differentiation

Conference Molecular Therapy · May 2016 Full text Cite

482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy

Conference Molecular Therapy · May 2016 Full text Cite

504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy

Conference Molecular Therapy · May 2016 Full text Cite

A Molecule-Scale View of the Structure and Specificity of the RNA-Guided Endonuclease Cas9

Conference Biophysical Journal · February 2016 Full text Cite

Anatomically-Shaped Tissue-Engineered Cartilage with Tunable and Inducible Anti-Inflammatory Capabilities

Conference TISSUE ENGINEERING PART A · September 1, 2015 Link to item Cite

CRISPRi Immunomodulation for Tissue Engineering/Stem Cell Therapies Targeting Intervertebral Disc Degeneration

Conference TISSUE ENGINEERING PART A · September 1, 2015 Link to item Cite

59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype

Conference Molecular Therapy · May 2015 Full text Cite

504. Targeted Genome Engineering of Induced Pluripotent Stem Cells To Produce Auto-Regulated Inflammation Resistance for Musculoskeletal Regenerative Medicine

Conference Molecular Therapy · May 2015 Full text Cite

485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements

Conference Molecular Therapy · May 2015 Full text Cite

397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy

Conference Molecular Therapy · May 2015 Full text Cite

Zinc finger nuclease-induced targeted integration of a glucose-6-phosphatase gene promotes survival in mice with glycogen storage disease type-IA

Conference MOLECULAR GENETICS AND METABOLISM · March 1, 2015 Link to item Cite

CRISPR/Cas9-Based Transcriptional Repressors for Control of Human Gene Expression

Conference MOLECULAR THERAPY · May 1, 2014 Link to item Cite

Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods

Conference MOLECULAR THERAPY · May 1, 2014 Link to item Cite

Biomaterial-Mediated Lentiviral Gene Delivery for Osteochondral Tissue Engineering

Conference MOLECULAR THERAPY · May 1, 2014 Link to item Cite

Modulating the Expression of the Cell Cycle Inhibitor p21 to Enhance Cartilage Tissue Engineering With iPSCs

Conference MOLECULAR THERAPY · May 1, 2014 Link to item Cite

CRISPR/Cas9-Based Transcriptional Activation of Endogenous Myod1 to Reprogram Murine Fibroblasts into Skeletal Myocytes

Conference MOLECULAR THERAPY · May 2014 Link to item Cite

Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors

Conference MOLECULAR THERAPY · June 1, 2013 Link to item Cite

Tunable expression of IL-1Ra in genetically modified mesenchymal stem cells for cartilage tissue engineering

Conference Osteoarthritis and Cartilage · April 2013 Full text Cite

Biomaterial-Mediated Gene Delivery for Tissue Engineering of Articular Cartilage

Conference MOLECULAR THERAPY · May 1, 2012 Link to item Cite