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Therapeutic applications of trans-splicing.

Accepted version
Peer-reviewed

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Type

Article

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Authors

Hong, Elizabeth M 
Ingemarsdotter, Carin K 
Lever, Andrew ML 

Abstract

BACKGROUND: RNA trans-splicing joins exons from different pre-mRNA transcripts to generate a chimeric product. Trans-splicing can also occur at the protein level, with split inteins mediating the ligation of separate gene products to generate a mature protein. SOURCES OF DATA: Comprehensive literature search of published research papers and reviews using Pubmed. AREAS OF AGREEMENT: Trans-splicing techniques have been used to target a wide range of diseases in both in vitro and in vivo models, resulting in RNA, protein and functional correction. AREAS OF CONTROVERSY: Off-target effects can lead to therapeutically undesirable consequences. In vivo efficacy is typically low, and delivery issues remain a challenge. GROWING POINTS: Trans-splicing provides a promising avenue for developing novel therapeutic approaches. However, much more research needs to be done before developing towards preclinical studies. AREAS TIMELY FOR DEVELOPING RESEARCH: Increasing trans-splicing efficacy and specificity by rational design, screening and competitive inhibition of endogenous cis-splicing.

Description

Keywords

trans-splicing, cancer, gene therapy, genetic disease, infectious disease, ribozyme-mediated trans-splicing, spliceosome-mediated RNA trans-splicing (SMaRT), split intein-mediated trans-splicing, Humans, Inteins, Proteins, Trans-Splicing

Journal Title

Br Med Bull

Conference Name

Journal ISSN

0007-1420
1471-8391

Volume Title

136

Publisher

Oxford University Press (OUP)

Rights

All rights reserved
Sponsorship
Cancer Research UK (25332)
Clinical Academic Reserve CRUK