AAV micro-dystrophin

AAV micro-dystrophin is a gene therapy modality designed to treat Duchenne muscular dystrophy (DMD) by delivering a functional, truncated version of the dystrophin gene via an adeno-associated virus (AAV) vector. While most AAV micro-dystrophin programs focus on restoring muscle function, specific research (such as that conducted at University College London) utilizes neuronal-targeted promoters to address the neurobehavioral and cognitive complications of DMD, including anxiety and intellectual disability. By restoring dystrophin interactors in the central nervous system, these therapies aim to ameliorate emotional reactivity and other brain-related phenotypes that are not addressed by muscle-centric treatments. Preclinical studies have demonstrated efficacy in restoring dystrophin interactors in the brain and improving emotional reactivity behaviors in mouse models following both intracerebroventricular and intravenous administration.

Other names

AAV.micro-dystrophinneuronal targeted AAV micro-dystrophin gene