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Article

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2005

Madocsai Csilla, Lim Sharlene R, Geib Till, Lam Bianca J, Hertel Klemens J

Abstract excerpt

Mutations in one of the duplicated survival of motor neuron (SMN) genes lead to the progressive loss of motor neurons and subsequent development of spinal muscular atrophy (SMA), a common, and usually fatal, hereditary disease. Homozygous absence of the telomeric copy (SMN1) correlates with development of SMA because differential splicing of the centromeric copy (SMN2) leads to exon 7 skipping and predominantly...

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Share to Cyclic AMP Response Element-Binding Protein
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Atrophy, Spinal

Share to Oligonucleotides, Antisense

Share to RNA, Small Nuclear

Share to SMN Complex Proteins

Share to Survival of Motor Neuron 1 Protein

Share to Survival of Motor Neuron 2 Protein

Source

Identifiers

PMID: 16226920
DOI: 10.1016/j.ymthe.2005.08.022

Genes

SMN2
SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

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Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs.PMID 16226920Read ↗

Spinal Muscular Atrophy: SMN2 Pre-mRNA Splicing Corrected by a U7 snRNA Derivative Carrying a Splicing Enhancer SequenceSemantic proximity Read ↗

Improvement of SMN2 Pre-mRNA Processing Mediated by Exon-Specific U1 Small Nuclear RNASemantic proximity Read ↗

Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophySemantic proximity Read ↗

Antisense Oligonucleotides for the Treatment of Spinal Muscular AtrophySemantic proximity Read ↗

Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA SplicingSemantic proximity Read ↗

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulationSemantic proximity Read ↗

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy.Semantic proximity Read ↗

Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the ExonSemantic proximity Read ↗

Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAsSemantic proximity Read ↗

Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing.Semantic proximity Read ↗

Targeting SR Proteins Improves SMN Expression in Spinal Muscular Atrophy CellsSemantic proximity Read ↗

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