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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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: | https://repository.cshl.edu/id/eprint/27459 |
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