High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed <i>SMN2</i> Splicing in Patient Fibroblasts

High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed <i>SMN2</i> Splicing in Patient Fibroblasts

Spinal muscular atrophy (SMA) is caused by <i>survival motor neuron 1 SMN1</i> deletion. The <i>survival motor neuron 2 (SMN2)</i> encodes the same protein as <i>SMN1</i> does, but it has a splicing defect of exon 7. Some antisense oligonucleotides (ASOs) have bee...

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Bibliographic Details
Main Authors: Yogik Onky Silvana Wijaya, Emma Tabe Eko Niba, Hisahide Nishio, Kentaro Okamoto, Hiroyuki Awano, Toshio Saito, Yasuhiro Takeshima, Masakazu Shinohara
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:Genes
Subjects:
spinal muscular atrophy
<i>SMN1</i>
<i>SMN2</i>
splicing
antisense oligonucleotide
cryptic exon
Online Access:https://www.mdpi.com/2073-4425/13/4/685

Internet

https://www.mdpi.com/2073-4425/13/4/685

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