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Article

Therapeutic activity of modified U1 core spliceosomal particles.

Nature communications - 4 Apr 2016

Rogalska Malgorzata Ewa, Tajnik Mojca, Licastro Danilo, Bussani Erica, Camparini Luca, Mattioli Chiara, Pagani Franco

Abstract excerpt

Modified U1 snRNAs bound to intronic sequences downstream of the 5' splice site correct exon skipping caused by different types of mutations. Here we evaluate the therapeutic activity and structural requirements of these exon-specific U1 snRNA (ExSpeU1) particles. In a severe spinal muscular atrophy, mouse model, ExSpeU1, introduced by germline transgenesis, increases SMN2 exon 7 inclusion, SMN protein production...

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Topics

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Share to Animals, Genetically Modified
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Atrophy, Spinal

Share to Nucleic Acid Conformation

Share to RNA, Small Nuclear

Share to RNA Splice Sites

Share to RNA Splicing

Share to Spliceosomes

Source

Open in PubMed Open full text

Identifiers

PMID: 27041075
DOI: 10.1038/ncomms11168
PMCID: PMC4822034

Genes

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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