Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy
羊膜腔内反义寡核苷酸治疗可改善脊髓性肌萎缩症临床前模型的表型
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.adv4656
Borges, Beltran; Brown, Stephen M; Chen, Wan-Jin; Clarke, Maria T; Herzeg, Akos; Park, Jae Hong; Ross, Joshua; Kong, Lingling; Denton, Madeline; Smith, Amy K; Lum, Tony; Zada, Fareha Moulana; Cordero, Marco; Gupta, Nalin; Cook, Sarah E; Murray, Heather; Matson, John; Klein, Stephanie; Bennett, C Frank; Krainer, Adrian R; MacKenzie, Tippi C; Sumner, Charlotte J