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The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy

06 June 25 Articles ، DystroBlog | Neuromuscular Disease Research & News ، Jeffrey Chamberlain AAV-mediated gene therapy for Duchenne muscular dystrophy ، micro-dystrophin constructs in DMD gene therapy ، safety and efficacy of AAV-based gene delivery in DMD ، immunological response to AAV in Duchenne gene therapy ، systemic delivery of AAV vectors in muscular dystrophy ، preclinical models for DMD gene therapy testing ، FDA-approved trials for AAV-DMD treatments ، long-term expression of micro-dystrophin in muscle tissue ، challenges in dosing and delivery of AAV in DMD ، CRISPR vs AAV gene therapy approaches in dystrophinopathies
The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy
This article reviews the evolution of AAV-mediated gene therapy for DMD, focusing on dystrophin structure, μDys engineering, systemic delivery strategies, clinical trial outcomes, and emerging solutions such as myotropic AAVs and gene editing platforms designed to overcome current limitations and enhance therapeutic efficacy.
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Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery

22 March 25 Articles ، Douglas Millay ، Christopher W Ward ، Jeffrey Chamberlain targeted gene therapy for Duchenne muscular dystrophy treatment ، engineered viruses delivering genes specifically to muscle tissue ، selective muscle cell targeting using Myomaker and Myomerger proteins ، pseudotyped lentiviral vectors for skeletal muscle gene therapy ، re-dosable viral vectors for safe and sustained gene expression ، fusion-protein-based gene delivery to regenerating muscle fibers ، immune-safe strategies for repeated muscle gene therapy injections ، muscle-specific gene therapy with minimized off-target effects ، restoring dystrophin expression in DMD using μDys gene delivery ، novel viral platforms for efficient and precise gene delivery to muscle
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery
This study introduces muscle-specific pseudotyped viruses using natural fusion proteins (Myomaker & Myomerger) to deliver gene therapy directly and selectively to muscle tissue—offering new hope for muscular diseases like DMD.
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