| Splicing therapeutics in SMN2 and APOB. | |
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MedLine Citation:
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PMID: 19330716 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based compounds that target key elements in the pre-mRNA to control splicing in the nucleus. In this review, recent developments in splicing therapeutics for the treatment of two specific diseases are described: correcting the alternative splicing of survival of motor neuron (SMN)2 pre-mRNA to compensate for the defective SMN1 gene in spinal muscular atrophy, and re-engineering the splicing of apolipoprotein B pre-mRNA to lower circulating cholesterol levels. |
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Authors:
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Bernard Khoo; Adrian R Krainer |
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Publication Detail:
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Type: Journal Article; Review |
Journal Detail:
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Title: Current opinion in molecular therapeutics Volume: 11 ISSN: 2040-3445 ISO Abbreviation: Curr. Opin. Mol. Ther. Publication Date: 2009 Apr |
Date Detail:
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Created Date: 2009-03-30 Completed Date: 2009-06-11 Revised Date: 2011-09-26 |
Medline Journal Info:
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Nlm Unique ID: 100891485 Medline TA: Curr Opin Mol Ther Country: England |
Other Details:
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Languages: eng Pagination: 108-15 Citation Subset: IM |
Affiliation:
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University College London Medical School, Department of Endocrinology, Hampstead Campus, London, UK. b.khoo@ucl.ac.uk |
Export Citation:
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| MeSH Terms | |
Descriptor/Qualifier:
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Anticholesteremic Agents
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therapeutic use* Apolipoproteins B / genetics* Exons / genetics Humans Models, Biological Muscular Atrophy, Spinal / drug therapy*, genetics Oligonucleotides, Antisense / genetics, therapeutic use* RNA Splicing / genetics* SMN Complex Proteins / genetics* Survival of Motor Neuron 2 Protein |
| Grant Support | |
ID/Acronym/Agency:
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R01 GM042699-21/GM/NIGMS NIH HHS |
| Chemical | |
Reg. No./Substance:
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0/Anticholesteremic Agents; 0/Apolipoproteins B; 0/Oligonucleotides, Antisense; 0/SMN Complex Proteins; 0/SMN2 protein, human; 0/Survival of Motor Neuron 2 Protein |
| Comments/Corrections | |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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