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Article

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.

Scientific reports - 30 Jun 2015

Nizzardo Monica, Simone Chiara, Dametti Sara, Salani Sabrina, Ulzi Gianna, Pagliarani Serena, Rizzo Federica, Frattini Emanuele, Pagani Franco, Bresolin Nereo, Comi Giacomo, Corti Stefania

Abstract excerpt

Spinal muscular atrophy (SMA) is a primary genetic cause of infant mortality due to mutations in the Survival Motor Neuron (SMN) 1 gene. No cure is available. Antisense oligonucleotides (ASOs) aimed at increasing SMN levels from the paralogous SMN2 gene represent a possible therapeutic strategy. Here, we tested in SMA human induced pluripotent stem cells (iPSCs) and iPSC-differentiated motor neurons, three...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 26123042
DOI: 10.1038/srep11746
PMCID: PMC4485234

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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