AAV9 microdystrophin vector

AAV9 microdystrophin vector is a gene therapy modality designed to treat Duchenne muscular dystrophy (DMD) by delivering a functional, truncated version of the dystrophin gene (microdystrophin) to muscle cells. It utilizes an adeno-associated virus serotype 9 (AAV9) vector, which has a high tropism for skeletal and cardiac muscle. Once inside the cell, the transgene expresses a microdystrophin protein that compensates for the lack of native dystrophin, helping to stabilize muscle cell membranes and prevent progressive muscle degeneration. Several companies have developed specific candidates using this platform, including Pfizer (fordadistrogene movaparvovec), Solid Biosciences (SGT-001, SGT-003), and Genethon.

Other names

AAV9-microdystrophinAAV-9-microdystrophinAAV 9-microdystrophinAAV9-uDysAAV-9-uDysAAV 9-uDysAAV9-mini-dystrophinAAV-9-mini-dystrophinAAV 9-mini-dystrophin