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Article

Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.

Clinical therapeutics - 1 Mar 2014

Nizzardo Monica, Simone Chiara, Salani Sabrina, Ruepp Marc-David, Rizzo Federica, Ruggieri Margherita, Zanetta Chiara, Brajkovic Simona, Moulton Hong M, Müehlemann Oliver, Bresolin Nereo, Comi Giacomo P, Corti Stefania

Abstract excerpt

BACKGROUND: Spinal muscular atrophy (SMA) is a fatal motor neuron disease of childhood that is caused by mutations in the SMN1 gene. Currently, no effective treatment is available. One possible therapeutic approach is the use of antisense oligos (ASOs) to redirect the splicing of the paralogous gene SMN2, thus increasing functional SMN protein production. Various ASOs with different chemical properties are...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Survival of Motor Neuron 2 Protein

Source

Identifiers

PMID: 24636820
DOI: 10.1016/j.clinthera.2014.02.004

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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