NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice

Published 2019 View Full Article

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Title
NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice
Authors
Keywords
spinal muscular atrophy, SMA, neuromuscular disorder, motor neuron disorder, neuromuscular junction, SMN1, SMN2, NCALD, modifier gene, ASO therapy
Journal
AMERICAN JOURNAL OF HUMAN GENETICS
Volume 105, Issue 1, Pages 221-230
Publisher
Elsevier BV
Online
2019-06-20
DOI
10.1016/j.ajhg.2019.05.008

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