Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

The severe childhood disease spinal muscular atrophy (SMA) arises from the homozygous loss of the survival motor neuron 1 gene (SMN1). A homologous gene potentially encoding an identical protein, SMN2 can partially compensate for the loss of SMN1; however, the exclusion of a critical exon in the cod...

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Bibliographic Details
Main Authors: Loren L. Flynn, Chalermchai Mitrpant, Ianthe L. Pitout, Sue Fletcher, Steve D. Wilton
Format: Article
Language:English
Published: Elsevier 2018-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
intron retention
antisense oligonucleotide
splice modification
Online Access:http://www.sciencedirect.com/science/article/pii/S216225311830012X

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http://www.sciencedirect.com/science/article/pii/S216225311830012X

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