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Article

Nusinersen in the Treatment of Spinal Muscular Atrophy.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2018

Goodkey Kara, Aslesh Tejal, Maruyama Rika, Yokota Toshifumi

Abstract excerpt

Spinal muscular atrophy (SMA) is one of the most common genetic causes of infantile death arising due to mutations in the SMN1 gene and the subsequent loss of motor neurons. With the discovery of the intronic splicing silencer N1 (ISS-N1) as a potential target for antisense therapy, several antisense oligonucleotides (ASOs) are being developed to include exon 7 in the final mRNA transcript of the SMN2 gene and...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 30171535
DOI: 10.1007/978-1-4939-8651-4_4

Genes

SMN1
SMN2

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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