AAV9-UFμDys1

AAV9-UFμDys1 is an adeno-associated virus serotype 9 (AAV9) gene therapy vector designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes an optimized expression cassette, UFμDys1, to deliver a functional microdystrophin transgene to striated muscle and cardiac tissues, aiming to compensate for the lack of endogenous dystrophin. Developed at the University of Florida, this vector is being studied in conjunction with immunosuppression strategies to mitigate immune responses to the AAV capsid, thereby potentially allowing for the treatment of patients with pre-existing anti-AAV antibodies and enabling redosing protocols. Preclinical studies in mdx mice have demonstrated significant improvements in muscle force and sustained microdystrophin expression.

Other names

AAV9-UF-microdystrophinAAV-9-UF-microdystrophinAAV 9-UF-microdystrophin