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Article

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.

Science translational medicine - 9 Jun 2010

Valori Chiara F, Ning Ke, Wyles Matthew, Mead Richard J, Grierson Andrew J, Shaw Pamela J, Azzouz Mimoun

Abstract excerpt

Spinal muscular atrophy is one of the most common genetic causes of death in childhood, and there is currently no effective treatment. The disease is caused by mutations in the survival motor neuron gene. Gene therapy aimed at restoring the protein encoded by this gene is a rational therapeutic approach to ameliorate the disease phenotype. We previously reported that intramuscular delivery of a lentiviral vector...

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Source

Identifiers

PMID: 20538619
DOI: 10.1126/scitranslmed.3000830

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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