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Article

Defining the therapeutic window in a severe animal model of spinal muscular atrophy.

Human molecular genetics - 1 Sept 2014

Robbins Kate L, Glascock Jacqueline J, Osman Erkan Y, Miller Madeline R, Lorson Christian L

Abstract excerpt

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by the loss of a single gene, Survival Motor Neuron-1 (SMN1). Administration of a self-complementary Adeno-Associated Virus vector expressing full-length SMN cDNA (scAAV-SMN) has proven an effective means to rescue the SMA phenotype in SMA mice, either by intravenous (IV) or intracerebroventricular (ICV) administration at very early time points....

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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: 24722206
DOI: 10.1093/hmg/ddu169
PMCID: PMC4119406

Genes

SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

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