AAV-Mediated Gene Transfer of a Novel Microdystrophin Ameliorates Pathology and Enhances Muscle Function in a Mouse Model of DMD This study evaluates preclinical efficacy of RGX-202, an AAV8-microdystrphin with extended CT-domain undergoing clinical investigations for Duchenne Muscular Dystrophy (NCT05693142), in mdx mice. Hist...

New #MolTherNucAcids paper: AAV-Mediated Gene Transfer of a Novel Microdystrophin Ameliorates Pathology and Enhances Muscle Function in a Mouse Model of DMD. #Myoblue tinyurl.com/mrxhvrhm

Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy This study uses a zebrafish model of Duchenne muscular dystrophy to identify an HDACi, SR-4370, that improves muscle structure and survival in dmd zebrafish. They show that HDACi that improve dmd zebr...

Congrats #maves_lab and colleagues for their new #MolTherNucAcids paper: Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy. #Myoblue tinyurl.com/2jacesdj

Congrats to @jyotijaiswal.bsky.social & colleagues for their new #MolTherNucAcids paper: A Combinatorial Oligonucleotide Therapy to Improve Dystrophin Restoration and Dystrophin-Deficient Muscle Health. #Myoblue tinyurl.com/bdm7xw83