Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease
在消失性白质疾病小鼠模型中评估AAV基因疗法的安全性和早期疗效
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2024.03.034
Jessica A Herstine ,Pi-Kai Chang ,Sergiy Chornyy ,Tamara J Stevenson ,Alex C Sunshine ,Ksenia Nokhrina ,Jessica Rediger ,Julia Wentz ,Tatyana A Vetter ,Erika Scholl ,Caleb Holaway ,Nettie K Pyne ,Anna Bratasz ,Stewart Yeoh ,Kevin M Flanigan ,Joshua L Bonkowsky ,Allison M Bradbury
