Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.

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Bibliographische Detailangaben
Veröffentlicht in:	Nature neuroscience 2017-04, Vol.20 (4), p.497-499
1. Verfasser:	Corey, David R
Format:	Artikel
Sprache:	eng
Schlagworte:	631/154/51/391 631/92/500 Animal Genetics and Genomics Animals Atrophy Behavioral Sciences Biological Techniques Biomedicine Care and treatment Cell culture Development and progression Diagnostic tests Disease Drug Discovery - methods Drug Discovery - trends Drug dosages Gene mutation Genes Genetic aspects Health aspects Humans Innovations Molecular targeted therapy Muscular Atrophy, Spinal - diagnosis Muscular Atrophy, Spinal - drug therapy Muscular Atrophy, Spinal - physiopathology Mutation Neurobiology Neurosciences news-and-views Nusinersen Oligonucleotides - therapeutic use Oligonucleotides, Antisense - therapeutic use Patients Proteins Psychological aspects Spinal muscular atrophy
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Beschreibung
Zusammenfassung:	Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.
ISSN:	1097-6256 1546-1726
DOI:	10.1038/nn.4508