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Article

Is RNA manipulation a viable therapy for spinal muscular atrophy?

Journal of the neurological sciences - 15 Dec 2009

Horne Christopher, Young Philip J

Abstract excerpt

Childhood spinal muscular atrophy (SMA) is an autosomal recessive disorder characterised by loss of the alpha motor neurones of the spinal cord. SMA is cause by mutations in the survival motor neuron (SMN) gene. There are two copies of the SMN gene: SMN1 and SMN2. The two genes differ by only 11 nucleotides at the genomic level. One of these is a C to T single nucleotide polymorphism (SNP) at position 6 in exon...

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Source

Identifiers

PMID: 19758605
DOI: 10.1016/j.jns.2009.08.055

Genes

SMN2
SMN1

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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