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 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.more
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery 22 March 25
ویروسهای پوشیدهشده با ترکیبکنندههای سلولی میوژنیک پستانداران، عضله اسکلتی را برای تحویل ژن هدف قرار میدهند 22 March 25
Recent developments and industry interest in gene therapy for duchenne muscular dystrophy 13 March 25
Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy 06 March 25
الیگونوکلئوتیدهای آنتی سنس کونژوگه با ترکیبات چربی دوست: سنتز و ارزیابی آزمایشگاهی پرش اگزون در دیستروفی عضلانی دوشن 06 March 25
In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy 06 March 25
غربالگری از طریق مدل سازی کامپیوتری بر اساس الگوریتم های پیش بینی به عنوان ابزار طراحی برای الیگونوکلئوتیدهای پرش اگزون در دیستروفی عضلانی دوشن 06 March 25