期刊
BRITISH MEDICAL BULLETIN
卷 136, 期 1, 页码 4-20出版社
OXFORD UNIV PRESS
DOI: 10.1093/bmb/ldaa028
关键词
trans-splicing; spliceosome-mediated RNA trans-splicing (SMaRT); ribozyme-mediated trans-splicing; split intein-mediated trans-splicing; genetic disease; infectious disease; cancer; gene therapy
资金
- Cancer Research UK [C63549/A25332]
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.
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