AAV6-CK8e-mDys5

AAV6-CK8e-mDys5 is an investigational adeno-associated virus (AAV) gene therapy construct designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes an AAV serotype 6 capsid to deliver a truncated micro-dystrophin transgene, known as micro-dystrophin 5 (mDys5 or μDys5), under the control of the muscle-specific CK8e (Creatine Kinase 8 enhancer) promoter. The μDys5 transgene is optimized for high expression and efficacy in both skeletal and cardiac muscle, aiming to restore a functional version of the dystrophin protein to the sarcolemma. While the AAV6-based construct is primarily used in preclinical research (such as in the Fiona/dko mouse model), the μDys5 transgene itself is the therapeutic component of clinical-stage candidates like SGT-001, developed by Solid Biosciences. Preclinical studies have demonstrated that this therapy can prevent cardiac pathology, maintain ejection fraction, and reduce inflammation and fibrosis in severe models of DMD cardiomyopathy.

Other names

micro-dystrophin 5 gene therapymicro-dystrophin5 gene therapymicro-dystrophin-5 gene therapyμDys5 gene therapy