AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial.
Lewis CJ, D'Souza P, Johnston JM, Acosta MT, Farmer C, Baker EH, Crowell A, Mojica Y, Rahman S, Joseph L, Hartman A, Vézina G, Quezado Z, Yousef MH, Luckett A, Vardar Z, Shazeeb MS, Corti M, Blackwood M, Coleman K, Thurm A, De Boever E, Gahl WA, Byrne BJ, Flotte TR, Jiang X, Gross AL, Keeler AM, Gray-Edwards H, Martin DR, Sena-Esteves M, Tifft CJ.
Lewis CJ, et al. Among authors: baker eh.
medRxiv [Preprint]. 2025 Jul 29:2025.07.28.25332074. doi: 10.1101/2025.07.28.25332074.
medRxiv. 2025.
PMID: 40766118
Free PMC article.
Updated.
Preprint.