Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. All patients have at least one copy of a paralog, SMN2, but a C-to-T transition in this gene results in exon 7 skipping in a majority of transcripts. Approved treatment for SMA i...

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Bibliographic Details
Main Authors: Audrey M. Winkelsas, Christopher Grunseich, George G. Harmison, Katarzyna Chwalenia, Carlo Rinaldi, Suzan M. Hammond, Kory Johnson, Melissa Bowerman, Sukrat Arya, Kevin Talbot, Matthew J. Wood, Kenneth H. Fischbeck
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotides
spinal muscular atrophy
5′ UTR
SMN2
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312030408X

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http://www.sciencedirect.com/science/article/pii/S216225312030408X

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