AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Murakami Y, Umeshita S, Imanishi K, Yoshioka Y, Ninomiya A, Sunabori T, Likhite S, Koike M, Meyer KC, Kinoshita T. Mol Ther Methods Clin Dev. 2024 Mar 14; 32(1):101176. Epub 2023 Dec 14.