Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford OX3 9DU, UK.
MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford OX1 3QX, UK.
Sci Transl Med. 2019 Sep 25;11(511). doi: 10.1126/scitranslmed.aay2069.
Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.
反义寡核苷酸疗法有效治疗脊髓性肌萎缩症，为采用该方法治疗其他神经疾病开辟了道路。
