AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models
AAV9 介导的 MYBPC3 基因治疗与优化的表达盒可增强 MYBPC3 心肌病模型的心脏功能和生存率
期刊:Nature Communications
影响因子:14.7
doi:10.1038/s41467-025-57481-7
Amara Greer-Short, Anna Greenwood, Elena C Leon, Tawny Neal Qureshi, Konor von Kraut, Justin Wong, Jonathan H Tsui, Christopher A Reid, Ze Cheng, Emilee Easter, Jin Yang, Jaclyn Ho, Stephanie Steltzer, Ana Budan, Marie Cho, Rishikesan Chandrakumar, Olga Cisne-Thompson, Charles Feathers, Tae Won Chun
