AAV9-U7snRNA

**AAV9-U7snRNA** is an adeno-associated virus serotype 9 based **gene therapy** platform for **Duchenne muscular dystrophy** that delivers engineered **U7 small nuclear RNA** exon-skipping cassettes to muscle after systemic administration. The therapy is designed to restore dystrophin expression by using modified U7snRNA antisense sequences to redirect splicing of **DMD** pre-mRNA and skip selected exons, thereby enabling production of a shorter but functional dystrophin protein. Reported implementations include **AAV9-U7ex51** for exon 51 skipping in preclinical models and **scAAV9.U7.ACCA** for exon 2 duplication-associated DMD, which has been evaluated clinically as an intravenous single-dose program developed at **Nationwide Children’s Hospital**.

Other names

AAV9-U7 small nuclear RNAAAV-9-U7 small nuclear RNAAAV 9-U7 small nuclear RNAAAV9-U7snRNA-mediated exon skippingAAV-9-U7snRNA-mediated exon skippingAAV 9-U7snRNA-mediated exon skipping