Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond

Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials

(2023) Jeffrey Chamberlain et al.   HUMAN GENE THERAPY

Dwarf Open Reading Frame (DWORF) Gene Therapy Ameliorated Duchenne Muscular Dystrophy Cardiomyopathy in Aged mdx Mice

(2023) Emily D. Morales et al.   Journal of the American Heart Association

Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies

(2023) Sina Fatehi et al.   HUMAN GENE THERAPY

Optogenetic Control of Muscles: Potential Uses and Limitations

(2023) E. Myfanwy Cohen et al.   HUMAN GENE THERAPY

The Future of Exon Skipping for Duchenne Muscular Dystrophy

(2023) Annemieke Aartsma-Rus   HUMAN GENE THERAPY

Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors

(2023) Jixin Liu et al.   HUMAN GENE THERAPY

CRISPR-Editing Therapy for Duchenne Muscular Dystrophy

(2023) Francesco Chemello et al.   HUMAN GENE THERAPY

Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies

(2023) Sandeep R.P. Kumar et al.   HUMAN GENE THERAPY

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

(2022) Audrey Bourdon et al.   GENE THERAPY

After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial

(2022) Alex Philippidis   HUMAN GENE THERAPY

Durability of transgene expression after rAAV gene therapy

(2022) Manish Muhuri et al.   MOLECULAR THERAPY

Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

(2022) Dileep R. Karri et al.   Molecular Therapy-Nucleic Acids

Fatal Thrombotic Microangiopathy Case following Adeno-Associated Viral SMN Gene Therapy

(2022) Julia Guillou et al.   Blood Advances

Microdystrophin Therapy Rescues Impaired Na Currents in Cardiac Purkinje Fibers From Dystrophin-Deficient Mdx Mice

(2022) Janine Ebner et al.   Circulation-Arrhythmia and Electrophysiology

Fantastic AAV Gene Therapy Vectors and How to Find Them—Random Diversification, Rational Design and Machine Learning

(2022) Jonas Becker et al.   Pathogens

Seroprevalence of AAV neutralizing antibodies in males with Duchenne muscular dystrophy

(2022) Sumit Verma et al.   HUMAN GENE THERAPY

The implication of hinge 1 and hinge 4 in micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2022) Lakmini P. Wasala et al.   HUMAN GENE THERAPY

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

(2022) Jihad El Andari et al.   Science Advances

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy

(2022) Karen Anthony et al.   HUMAN GENE THERAPY

Brother of Cure Rare Disease CEO Dies in Trial of Duchenne Muscular Dystrophy Therapy

(2022) Alex Philippidis   HUMAN GENE THERAPY

Life-long outcomes of systemic AAV micro-dystrophin gene therapy in a murine Duchenne muscular dystrophy model

(2022) Nalinda Wasala et al.   HUMAN GENE THERAPY

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

(2021) Mohammadsharif Tabebordbar et al.   CELL

Fourth Boy Dies in Clinical Trial of Astellas' AT132

(2021) Alex Philippidis   HUMAN GENE THERAPY

The 2022 version of the gene table of neuromuscular disorders (nuclear genome)

(2021) Enzo Cohen et al.   NEUROMUSCULAR DISORDERS

Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

(2021) Chady H. Hakim et al.   Nature Communications

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

(2020) Nalinda B. Wasala et al.   MOLECULAR THERAPY

AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

(2020) Ang Li et al.   MOLECULAR THERAPY

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

(2020) Jennifer B. Kwon et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

(2019) Michael E. Nance et al.   MOLECULAR THERAPY

Micro-utrophin Therapy for Duchenne Muscular Dystrophy

(2019) Dongsheng Duan   MOLECULAR THERAPY

Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice

(2017) Antanina Voit et al.   Nature Communications

Systemic delivery of adeno-associated viral vectors

(2016) Dongsheng Duan   Current Opinion in Virology

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

(2015) J. W. McGreevy et al.   Disease Models & Mechanisms

Anti-Dystrophin T Cell Responses in Duchenne Muscular Dystrophy: Prevalence and a Glucocorticoid Treatment Effect

(2013) Kevin M. Flanigan et al.   Human Gene Therapy Methods