Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

(2019) Yi-Li Min et al.   Science Advances

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Eteplirsen treatment for Duchenne muscular dystrophy

(2018) Jay S. Charleston et al.   NEUROLOGY

Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing

(2018) Yu Zhang et al.   PHYSIOLOGICAL REVIEWS

CRISPR/Cas9 cleavages in budding yeast reveal templated insertions and strand-specific insertion/deletion profiles

(2018) Brenda R. Lemos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

(2018) Chengzu Long et al.   Science Advances

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

(2017) Eric R. Pozsgai et al.   MOLECULAR THERAPY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy

(2017) Pei Zhu et al.   Molecular Therapy-Nucleic Acids

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

(2017) Yu Zhang et al.   Science Advances

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Cas9-catalyzed DNA Cleavage Generates Staggered Ends: Evidence from Molecular Dynamics Simulations

(2016) Zhicheng Zuo et al.   Scientific Reports

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

Dynamics of Cell Generation and Turnover in the Human Heart

(2015) Olaf Bergmann et al.   CELL

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

(2014) R. Jude Samulski et al.   Annual Review of Virology

Satellite Cells and the Muscle Stem Cell Niche

(2013) Hang Yin et al.   PHYSIOLOGICAL REVIEWS

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

(2011) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

The DNA Damage Response: Making It Safe to Play with Knives

(2010) Alberto Ciccia et al.   MOLECULAR CELL

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

(2010) Joe N Kornegay et al.   MOLECULAR THERAPY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Evidence for Cardiomyocyte Renewal in Humans

(2009) Olaf Bergmann et al.   SCIENCE

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY