• Elizabeth M Hong, Carin K Ingemarsdotter, and LeverAndrew M LAMLDepartment of Medicine, University of Cambridge, Addenbrooke's Hospital, Hills Road, Cambridge CB2 2QQ, UK..
• Department of Medicine, University of Cambridge, Addenbrooke's Hospital, Hills Road, Cambridge CB2 2QQ, UK.
• Br. Med. Bull. 2020 Dec 15; 136 (1): 4204-20.

BackgroundRNA 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 DataComprehensive literature search of published research papers and reviews using Pubmed.Areas Of AgreementTrans-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 ControversyOff-target effects can lead to therapeutically undesirable consequences. In vivo efficacy is typically low, and delivery issues remain a challenge.Growing PointsTrans-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 ResearchIncreasing trans-splicing efficacy and specificity by rational design, screening and competitive inhibition of endogenous cis-splicing.© The Author(s) 2020. Published by Oxford University Press. All rights reserved. For Permissions, please email: journals.permissions@oup.com.

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