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Article

RNA in spinal muscular atrophy: therapeutic implications of targeting.

Expert opinion on therapeutic targets - 1 Aug 2020

Singh Ravindra N, Seo Joonbae, Singh Natalia N

Abstract excerpt

INTRODUCTION: Spinal muscular atrophy (SMA) is caused by low levels of the Survival Motor Neuron (SMN) protein due to deletions of or mutations in the SMN1 gene. Humans carry another nearly identical gene, SMN2, which mostly produces a truncated and less stable protein SMNΔ7 due to predominant skipping of exon 7. Elevation of SMN upon correction of SMN2 exon 7 splicing and gene therapy have been proven to be the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 32538213
DOI: 10.1080/14728222.2020.1783241
PMCID: PMC7529864

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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