AAV.cc47-Slc3a1

AAV.cc47-Slc3a1 is a preclinical adeno-associated virus (AAV) gene therapy being developed by Vanderbilt University and Duke University for the treatment of type A cystinuria [SA-PO647]. It utilizes a novel, cross-species compatible AAV capsid, AAV.cc47, which is engineered to selectively target proximal tubule epithelial cells (PTECs) in the kidney. The vector delivers the Slc3a1 transgene, which encodes the rBAT protein, a crucial subunit of the renal cystine transporter. In type A cystinuria, deficiency of rBAT leads to impaired cystine transport and reabsorption, resulting in recurrent cystine stones, urinary obstruction, and chronic kidney disease [SA-PO647]. By delivering Slc3a1, AAV.cc47-Slc3a1 aims to restore functional rBAT expression and rescue cystine transport in the kidney [SA-PO647].