High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy
来自 ASPIRO 的高通量转录组分析,ASPIRO 是一项针对 X 连锁肌管性肌病基因替代疗法的 1/2/3 期研究。
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2023.08.008
Andreoletti, Gaia; Romano, Oriana; Chou, Hsin-Jung; Sefid-Dashti, Mahjoubeh J; Grilli, Andrea; Chen, Clarice; Lakshman, Neema; Purushothaman, Pravin; Varfaj, Fatbardha; Mavilio, Fulvio; Bicciato, Silvio; Urbinati, Fabrizia