adeno-associated virus serotype 9 activation-induced cytidine deaminase cytosine base editor

Developed by GenAssist Ltd, this is an all-in-one adeno-associated virus serotype 9 (AAV9) gene therapy utilizing a cytosine base editor (CBE) for the treatment of Duchenne Muscular Dystrophy (DMD). The system employs a minimized AID (activation-induced cytidine deaminase) fused to a saCas9 protein, engineered to fit within the 4.7kb packaging capacity of a single AAV vector. It is designed to induce exon skipping (e.g., exon 50, 51, or 53) by precisely editing DNA sequences to restore the dystrophin reading frame. Preclinical studies in iPSC myotubes and humanized mouse models have demonstrated effective DNA editing and exon skipping, although initial animal studies noted low vector distribution in heart and muscle tissues, prompting the consideration of more myotropic AAV variants.

Other names

AID-based cytosine base editorAID CBE