Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity
AR 亚型 2 基因治疗通过调节 AR 转录活性挽救脊髓和延髓肌萎缩表型
期刊:Science Advances
影响因子:11.7
doi:10.1126/sciadv.abi6896
Wooi F Lim, Mitra Forouhan, Thomas C Roberts, Jesse Dabney, Ruth Ellerington, Alfina A Speciale, Raquel Manzano, Maria Lieto, Gavinda Sangha, Subhashis Banerjee, Mariana Conceição, Lara Cravo, Annabelle Biscans, Loïc Roux, Naemeh Pourshafie, Christopher Grunseich, Stephanie Duguez, Anastasia Khvorov