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Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.

More information Original publication

DOI

10.1126/scitranslmed.aay2069

Type

Journal article

Publication Date

2019-09-25T00:00:00+00:00

Volume

11

Keywords

Animals, Humans, Nerve Degeneration, Nervous System Diseases, Oligonucleotides, Antisense, RNA