Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome.
Ding WY, Kuzmuk V, Hunter S, Lay A, Hayes B, Beesley M, Rollason R, Hurcombe JA, Barrington F, Masson C, et al. Sci Transl Med. 2023 Aug 9; 15(708):eabc8226. Epub 2023 Aug 9.