Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
Author:
Affiliation:
1. Academic Neurology Unit, Department of Neuroscience, University of Sheffield, Beech Hill Road, S10 2RX Sheffield, UK.
Abstract
Publisher
American Association for the Advancement of Science (AAAS)
Subject
General Medicine
Reference29 articles.
1. Spinal muscular atrophy
2. Identification and characterization of a spinal muscular atrophy-determining gene
3. Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1
4. Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos
5. SMNΔ7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN
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