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Article

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 5 Aug 2020

Besse Aurore, Astord Stephanie, Marais Thibaut, Roda Marianne, Giroux Benoit, Lejeune François-Xavier, Relaix Frederic, Smeriglio Piera, Barkats Martine, Biferi Maria Grazia

Abstract excerpt

Spinal muscular atrophy (SMA) is a neuromuscular disease mainly caused by mutations or deletions in the survival of motor neuron 1 (SMN1) gene and characterized by the degeneration of motor neurons and progressive muscle weakness. A viable therapeutic approach for SMA patients is a gene replacement strategy that restores functional SMN expression using adeno-associated virus serotype 9 (AAV9) vectors. Currently,...

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Identifiers

PMID: 32470325
DOI: 10.1016/j.ymthe.2020.05.011
PMCID: PMC7403319

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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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