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Article

Combination of SMN <i>Trans</i> -Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular Atrophy

30 Aug 2010

Abstract excerpt

Spinal muscular atrophy (SMA), a neurodegenerative disease, is the second most common genetic disorder and the leading genetic cause of infantile death. SMA arises from the loss of Survival Motor Neuron-1 (SMN1), leading to degeneration of lower motor neurons and, consequently, the atrophy of voluntary muscles. A duplicated copy gene called SMN2 exists in humans. SMN2 is unable to fully compensate for the loss of...

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PMID: 20804424
DOI: 10.1089/hum.2010.114

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Combination of SMN <i>Trans</i> -Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular AtrophyPMID 20804424Read ↗

<i>Trans</i>-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular AtrophySemantic proximity Read ↗

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 TranscriptsSemantic proximity Read ↗

Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA SplicingSemantic proximity Read ↗

Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular AtrophySemantic proximity Read ↗

A Negatively Acting Bifunctional RNA Increases <i>Survival Motor Neuron</i> Both <i>In Vitro</i> and <i>In Vivo</i>Semantic proximity Read ↗

Decreasing Disease Severity in Symptomatic, <i>Smn</i> <sup>−/−</sup> ; <i>SMN2</i> <sup>+/+</sup> , Spinal Muscular Atrophy Mice Following scAAV9-SMN DeliverySemantic proximity Read ↗

Advances in Therapeutic Development for Spinal Muscular AtrophySemantic proximity Read ↗

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophySemantic proximity Read ↗

Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral VectorSemantic proximity Read ↗

Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and ChallengesSemantic proximity Read ↗

Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.Semantic proximity Read ↗

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouseSemantic proximity Read ↗

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