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Article

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.

Genes & development - 1 Feb 2015

Hua Yimin, Liu Ying Hsiu, Sahashi Kentaro, Rigo Frank, Bennett C Frank, Krainer Adrian R

Abstract excerpt

Survival of motor neuron (SMN) deficiency causes spinal muscular atrophy (SMA), but the pathogenesis mechanisms remain elusive. Restoring SMN in motor neurons only partially rescues SMA in mouse models, although it is thought to be therapeutically essential. Here, we address the relative importance of SMN restoration in the central nervous system (CNS) versus peripheral tissues in mouse models using a therapeutic...

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Share to Central Nervous System
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Mice, Transgenic

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Share to Muscular Atrophy, Spinal

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Source

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Identifiers

PMID: 25583329
DOI: 10.1101/gad.256644.114
PMCID: PMC4318145

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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