rAAVrh74.MCK.GALGT2

rAAVrh74.MCK.GALGT2 is a gene therapy product designed to deliver the GALGT2 gene (also known as B4GALNT2) to muscle cells using a viral vector (rAAVrh74). This therapy aims to compensate for the missing dystrophin protein in DMD patients by encouraging overproduction of proteins that stabilize muscle cell membranes and improve muscle function. Unlike other gene therapies that deliver portions of the DMD gene, this surrogate gene therapy introduces the GALGT2 gene with the goal of inducing production of proteins that inhibit muscular dystrophy progression.

Other names

GALGT2 gene therapy programGALGT-2 gene therapy programGALGT 2 gene therapy program