Splicing therapeutics in SMN2 and APOB

Khoo, B., Krainer, A. R. (April 2009) Splicing therapeutics in SMN2 and APOB. Current Opinion in Molecular Therapeutics, 11 (2). pp. 108-115. ISSN 1464-8431

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URL: http://www.ncbi.nlm.nih.gov/pubmed/19330716

Abstract

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.

Item Type: Paper
Uncontrolled Keywords: Antisense oligonucleotide APOB apolipoprotein B cholesterol RNA splicing SMN2 spinal muscular atrophy SPINAL MUSCULAR-ATROPHY SURVIVAL-MOTOR-NEURON MESSENGER-RNA ANTISENSE OLIGONUCLEOTIDES GENE-EXPRESSION PREMESSENGER RNA CRITICAL EXON IN-VITRO PROTEIN ENHANCER
Subjects: bioinformatics
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification
therapies > gene therapy
bioinformatics > genomics and proteomics > genetics & nucleic acid processing
bioinformatics > genomics and proteomics
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > RNA splicing
CSHL Authors:
Communities: CSHL labs > Krainer lab
Depositing User: Matt Covey
Date: April 2009
Date Deposited: 19 Feb 2013 17:56
Last Modified: 19 Feb 2013 17:56
PMCID: PMC3140428
Related URLs:
URI: http://repository.cshl.edu/id/eprint/27459

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