Gene Therapy Rescues Muscle, Eases Defects in Mice With Severe CMT2D

Gene Therapy Rescues Muscle, Eases Defects in Mice With Severe CMT2D

313716

Gene Therapy Rescues Muscle, Eases Defects in Mice With Severe CMT2D

A gene therapy delivering instructions to produce the neurotrophin-3 (NT-3) protein to muscle cells improved muscle function and eased defects in nerve and muscle structure of a mouse model of severe Charcot-Marie-Tooth disease type 2D (CMT2D), a study found. These results support an NT-3 gene therapy as a potential treatment approach for CMT2D and potentially for other CMT subtypes, its researchers wrote. The study, “AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice,” was published…

You must be logged in to read/download the full post.