First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Raymond Y. Wang, Tammam Beydoun, Nina Movsesyan, Shih-Hsin Kan, Mery Taylor, Winnie Stockton, Igor Nestrasil, Bryan Pukenas, Paulo Falabella, Yoonjin Cho, Marie-Laure Névoret
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research