Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.
Thomsen G, Kaspar A, Ferraiuolo L, Chu B, Garcia VJ, Weiss R, Slaiwa T, Casanova-Vallve N, Cano R, Hurley E, Julazadeh H, Bautista H, Renteria A, Lee M, Rollins J, McAlonis-Downes M, Arnold WD, TreDenick T, Hsu R, Bayless LE, Kozial C, Forespring C, Molony M, Fraser M, Zhou J, Yao J, Bremner SN, Ward SR, Schenk S, Yoshikawa T, Moriyama A, Tsusaki H, Varma S, Kruger M, Stahl M, Kaspar B.
Thomsen G, et al. Among authors: yao j.
Mol Ther Adv. 2026 Feb 20;34(1):201707. doi: 10.1016/j.omta.2026.201707. eCollection 2026 Mar 12.
Mol Ther Adv. 2026.
PMID: 42137291
Free PMC article.