Therapeutic applications of trans-splicing

AbstractBackgroundRNAtrans-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 bothin vitro andin 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 researchIncreasingtrans-splicing efficacy and specificity by rational design, screening and competitive inhibition of endogenouscis-splicing.
Source: British Medical Bulletin - Category: General Medicine Source Type: research