Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations
基因疗法治疗由GUCY2D突变引起的儿童失明后,安全性和疗效均有所提高
期刊:iScience
影响因子:4.1
doi:10.1016/j.isci.2021.102409
Jacobson, Samuel G; Cideciyan, Artur V; Ho, Allen C; Peshenko, Igor V; Garafalo, Alexandra V; Roman, Alejandro J; Sumaroka, Alexander; Wu, Vivian; Krishnan, Arun K; Sheplock, Rebecca; Boye, Sanford L; Cheang, Bee-Lin; Davidson, Vanessa; O'Riordan, Catherine R; Dizhoor, Alexander M; Boye, Shannon E