Gene Therapy Seen to Rescue Motor, Nerve Defects in CMTX1 Mouse Model

Gene Therapy Seen to Rescue Motor, Nerve Defects in CMTX1 Mouse Model

287140

Gene Therapy Seen to Rescue Motor, Nerve Defects in CMTX1 Mouse Model

Delivering a healthy neurotrophin-3 (NT-3) gene to muscle cells in a mouse model of Charcot-Marie-Tooth disease type X1 (CMTX1) prevented motor function decline and eased defects in nerve and muscle structure, a study found. These results support an NT-3 gene therapy as a potential treatment approach for this X-linked form of CMT and for other subtypes marked by problems with myelination. The study, “AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1,” was published in…

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