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Splicing therapeutics in SMN2 and APOB

Khoo, B; Krainer, AR; (2009) Splicing therapeutics in SMN2 and APOB. Current Opinion in Molecular Therapeutics , 11 (2) 108 - 115. Green open access

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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.

Type: Article
Title: Splicing therapeutics in SMN2 and APOB
Open access status: An open access version is available from UCL Discovery
Publisher version: http://ip-science.thomsonreuters.com/
Language: English
Additional information: Journal ceased publication
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
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Metabolism and Experi Therapeutics
URI: https://discovery.ucl.ac.uk/id/eprint/111955
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