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Article

Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.

Human molecular genetics - 1 Mar 2016

Feng Zhihua, Ling Karen K Y, Zhao Xin, Zhou Chunyi, Karp Gary, Welch Ellen M, Naryshkin Nikolai, Ratni Hasane, Chen Karen S, Metzger Friedrich, Paushkin Sergey, Weetall Marla, Ko Chien-Ping

Abstract excerpt

Spinal muscular atrophy (SMA) is a genetic disease characterized by atrophy of muscle and loss of spinal motor neurons. SMA is caused by deletion or mutation of the survival motor neuron 1 (SMN1) gene, and the nearly identical SMN2 gene fails to generate adequate levels of functional SMN protein due to a splicing defect. Currently, several therapeutics targeted to increase SMN protein are in clinical trials. An...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 26758873
DOI: 10.1093/hmg/ddv629

Genes

SMN2
SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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