Nucleic Acid-Based Therapeutic Approach for Spinal and Bulbar Muscular Atrophy and Related Neurological Disorders.

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作者： Tomoki,Hirunagi ^[1] ; Kentaro,Sahashi ^[1] ; Katherine G,Meilleur ^[2] ; Masahisa,Katsuno ^[1]

作者单位： Department of Neurology, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan. ^[1] Research and Clinical Development, Neuromuscular Development Unit, Biogen, 300, Binney Street, Cambridge, MA 02142, USA. ^[2] Department of Clinical Research Education, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan. ^[3]

关键词 antisense oligonucleotide motor neuron disease nucleic acid therapeutics polyglutamine disease small interference RNA spinal and bulbar muscular atrophy

主题词动物(Animals);延髓-脊髓肌萎缩症, X连锁(Bulbo-Spinal Atrophy, X-Linked);人类(Humans);神经系统疾病(Nervous System Diseases);寡核苷酸类, 反义(Oligonucleotides, Antisense);RNA, 小分子干扰(RNA, Small Interfering)

DOI 10.3390/genes13010109

PMID 35052449

发布时间 2022-02-22

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