AAV.U7snRNA exon 17 skipping vector

AAV.U7snRNA exon 17 skipping vector is a preclinical adeno-associated virus (AAV)-based gene therapy vector designed for the treatment of Duchenne muscular dystrophy (DMD). Developed at the Jerry R. Mendell Center for Gene Therapy at Nationwide Children's Hospital, the vector delivers a modified, non-coding U7 small nuclear RNA (U7snRNA) containing antisense sequences that target the splice acceptor, exonic splicing enhancers, or splice donor sites of DMD exon 17. By masking these splicing signals, the therapy induces exon 17 skipping during pre-mRNA splicing, restoring the reading frame of the DMD gene and enabling the production of an internally deleted but functional dystrophin protein in patients with amenable mutations.