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Therapeutic applications of trans-splicing
Elizabeth M Hong1, Carin K Ingemarsdotter1, Andrew M L Lever1
1Department of Medicine, University of Cambridge, Addenbrooke's Hospital, Hills Road, Cambridge CB2 2QQ, UK.
British Medical Bulletin
|October 3, 2020
Summary
RNA trans-splicing offers a novel therapeutic strategy by joining RNA or protein molecules. While promising for disease treatment, further research is needed to overcome challenges in efficacy and delivery.
Area of Science:
- Molecular Biology
- Gene Therapy
- Biochemistry
Background:
- RNA trans-splicing ligates exons from separate pre-mRNAs into a chimeric product.
- Protein-level trans-splicing utilizes split inteins for gene product ligation.
Purpose of the Study:
- To review the applications and challenges of trans-splicing techniques in therapeutic development.
- To identify areas for future research to advance trans-splicing therapies.
Main Methods:
- A comprehensive literature search was conducted using PubMed.
- Included published research papers and review articles on trans-splicing.
Main Results:
- Trans-splicing has demonstrated success in RNA, protein, and functional correction in vitro and in vivo disease models.
- Key challenges include low in vivo efficacy, delivery issues, and potential off-target effects.
Conclusions:
- Trans-splicing presents a promising therapeutic avenue for various diseases.
- Further research is essential to enhance efficacy, specificity, and address delivery challenges before preclinical studies.
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