Safety, efficacy, and distal nerve Schwann cell biodistribution in mice and NHPs to support translation of AAV9 RNAi therapy for CMT1A.
在小鼠和非人灵长类动物中评估 AAV9 RNAi 疗法治疗 CMT1A 的安全性、有效性和远端神经雪旺细胞生物分布，以支持其转化应用。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2026.102881
Stavrou Marina, Wallace Lindsay M, Thangaraj Merlin P, Taylor Noah K, Kagiava Alexia, Papacharalambous Revekka, McAllister Cynthia, Zender Gloria, Saad Nizar Y, Bayazit M Bilal, Heslegrave Amanda, Tryfonos Christina, Richter Jan, Zetterberg Henrik, Price Brian, Salzman Rachel, Kleopa Kleopas A, Harper Scott Q
