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Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMNDOI 10.1016/j.yneu.2011.05.043
The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophySemantic proximity
Advances in Therapeutic Development for Spinal Muscular AtrophySemantic proximity
Decreasing Disease Severity in Symptomatic, <i>Smn</i> <sup>−/−</sup> ; <i>SMN2</i> <sup>+/+</sup> , Spinal Muscular Atrophy Mice Following scAAV9-SMN DeliverySemantic proximity
A large animal model of spinal muscular atrophy and correction of phenotype.Semantic proximity
Spinal muscular atrophy: from tissue specificity to therapeutic strategiesSemantic proximity
Tissue-specific models of spinal muscular atrophy confirm a critical role of SMN in motor neurons from embryonic to adult stagesSemantic proximity
Generation of a tamoxifen inducible SMN mouse for temporal SMN replacement.Semantic proximity
Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.Semantic proximity
Animal models of spinal muscular atrophy.Semantic proximity
Update on gene and stem cell therapy approaches for spinal muscular atrophy.Semantic proximity
CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophySemantic proximity
Muscle: an independent contributor to the neuromuscular spinal muscular atrophy disease phenotype.Semantic proximity
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