Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results
前颗粒蛋白 AAV 基因治疗额颞叶痴呆:转化研究和 1/2 期试验中期结果
期刊:Nature Medicine
影响因子:58.7
doi:10.1038/s41591-024-02973-0
Jeffrey Sevigny, Olga Uspenskaya, Laura Dean Heckman, Li Chin Wong, Daniel A Hatch, Ambika Tewari, Rik Vandenberghe, David J Irwin, Dario Saracino, Isabelle Le Ber, Rebekah Ahmed, Jonathan D Rohrer, Adam L Boxer, Sebastian Boland, Patricia Sheehan, Alissa Brandes, Suzanne R Burstein, Benjamin M Shyk
信号转导
神经
阿尔茨海默症
Translational Control
Donkey
IgG