adeno-associated viral vector serotype 8 containing the 3'-MYO7A gene coding sequence

This adeno-associated viral (AAV) vector of serotype 8 is a critical component of the **AAVB-039** dual-vector gene therapy system developed by **Aavantgarde Bio**. It contains the 3' portion of the **MYO7A** gene coding sequence. Usher syndrome type 1B (USH1B) is caused by mutations in the *MYO7A* gene, which encodes the Myosin VIIA protein essential for the structural integrity of hair cells in the inner ear and photoreceptors in the retina. Because the *MYO7A* cDNA (~6.7 kb) exceeds the standard packaging capacity of a single AAV vector (~4.7 kb), Aavantgarde Bio utilizes a dual-vector approach. This 3' vector, when co-delivered with a corresponding 5' vector, enables the reconstitution of the full-length Myosin VIIA protein in retinal cells through protein trans-splicing mediated by inteins. The therapy is primarily indicated for retinitis pigmentosa associated with USH1B and is administered via subretinal injection to target the retinal pigment epithelium and photoreceptors.

Other names

AAV8-3'-MYO7A3'-MYO7A AAV8 vectorAAV8-intein-CAAV-8-intein-CAAV 8-intein-C