Science Translational Medicine· Nikki M. McCormack, Stephanie Lee-Diaz, Theodore R. Reyes, Marshall W. Hogarth, Jada L. Covington, Camryn A. Bragg, Zarek O. Harper, Thomas Diehn, James S. Novak, Christine L. Halbert, Jeffrey S. Chamberlain, David L. Mack, Martin K. Childers, Jyoti K. Jaiswal, Hichem Tasfaout·· 15 小时前评分61
分割内含肽与肌亲和性AAV载体实现dysferlin病临床前基因治疗
Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy
导读
Science Translational Medicine 发表的研究表明，使用分割内含肽（split intein）与肌亲和性AAV载体可在临床前模型中实现对dysferlin病的有效基因治疗。原文仅提供标题，完整摘要未随feed给出。
来源：Science Translational Medicine · science.org