Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
全身性肽介导的寡核苷酸疗法可改善脊髓性肌萎缩症患者的长期生存率
期刊:Proceedings of the National Academy of Sciences of the United States of America
影响因子:9.1
doi:10.1073/pnas.1605731113
Hammond, Suzan M; Hazell, Gareth; Shabanpoor, Fazel; Saleh, Amer F; Bowerman, Melissa; Sleigh, James N; Meijboom, Katharina E; Zhou, Haiyan; Muntoni, Francesco; Talbot, Kevin; Gait, Michael J; Wood, Matthew J A