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Article

Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA Splicing

5 Jun 2007

Abstract excerpt

Spinal muscular atrophy (SMA) is caused by loss of survival motor neuron-1 (SMN1). A nearly identical copy gene called SMN2 is present in all SMA patients; however SMN2 produces low levels of functional protein due to alternative splicing. Recently a therapeutic approach has been developed referred to as trans-splicing. Conceptually, this strategy relies upon pre-messenger RNA (pre-mRNA) splicing occurring...

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Identifiers

PMID: 17551501
DOI: 10.1038/sj.mt.6300222

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Related research

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Research trail→

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Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA SplicingPMID 17551501Read ↗

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

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

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

Spinal Muscular Atrophy: SMN2 Pre-mRNA Splicing Corrected by a U7 snRNA Derivative Carrying a Splicing Enhancer SequenceSemantic proximity Read ↗

Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAsSemantic proximity Read ↗

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 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 ↗

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 ↗

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs.Semantic proximity Read ↗

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulationSemantic proximity Read ↗

A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype.Semantic proximity Read ↗

RNA in spinal muscular atrophy: therapeutic implications of targeting.Semantic proximity Read ↗

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