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10.1002/wrna.1347 http://scihub22266oqcxt.onion/10.1002/wrna.1347 C5071737!5071737!27018401     free     free     free Deprecated: Implicit conversion from float 213.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Wiley+Interdiscip+Rev+RNA 2016 ; 7 (4): 487-98 Nephropedia Template TP {{tp|p=27018401|t=2016. mRNAtrans?splicing in gene therapy for genetic diseases |pdf=|usr=}}{{27018401}} gab.com Text mRNAtrans splicing in gene therapy for genetic diseases JO: Wiley Interdiscip Rev RNA http://www.kidney.de/pget.php?&tw=1&pmid=27018401 #FOAvip Spliceosome?mediated RNAtrans?splicing, or SMaRT, is a promising strategy to design innovative gene therapy solutions for currently intractable genetic diseases. SMaRT relies on the correction of mutations at the post?transcriptional level by modifying the mRNA sequence. To achieve this, an exogenous RNA is introduced into the target cell, usually by means of gene transfer, to induce a splice event in trans between the exogenous RNA and the target endogenous pre?mRNA. This produces a chimeric mRNA composed partly of exons of the latter, and partly of exons of the former, encoding a sequence free of mutations. The principal challenge of SMaRT technology is to achieve a reaction as complete as possible, i.e., resulting in 100% repairing of the endogenous mRNA target. The proof of concept of SMaRT feasibility has already been established in several models of genetic diseases caused by recessive mutations. In such cases, in fact, the repair of only a portion of the mutant mRNA pool may be sufficient to obtain a significant therapeutic effect. However in the case of dominant mutations, the target cell must be freed from the majority of mutant mRNA copies, requiring a highly efficient trans?splicing reaction. This likely explains why only a few examples of SMaRT approaches targeting dominant mutations are reported in the literature. In this review, we explain in details the mechanism of trans?splicing, review the different strategies that are under evaluation to lead to efficient trans?splicing, and discuss the advantages and limitations of SMaRT. WIREs RNA 2016, 7:487?498. doi: 10.1002/wrna.1347For further resources related to this article, please visit the WIREs website. Twit Text FOAVip mRNAtrans splicing in gene therapy for genetic diseases ????Wiley Interdiscip Rev RNA http://www.kidney.de/pget.php?&tw=1&pmid=27018401 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27018401 #FOAvip English Wikipedia <ref>{{Cite pmid|27018401}}</ref> mRNAtrans?splicing in gene therapy for genetic diseases #MMPMID27018401 Berger A; Maire S; Gaillard M; Sahel J; Hantraye P; Bemelmans A Wiley Interdiscip Rev RNA 2016[Jul]; 7 (4): 487-98 PMID27018401show ga Spliceosome?mediated RNAtrans?splicing, or SMaRT, is a promising strategy to design innovative gene therapy solutions for currently intractable genetic diseases. SMaRT relies on the correction of mutations at the post?transcriptional level by modifying the mRNA sequence. To achieve this, an exogenous RNA is introduced into the target cell, usually by means of gene transfer, to induce a splice event in trans between the exogenous RNA and the target endogenous pre?mRNA. This produces a chimeric mRNA composed partly of exons of the latter, and partly of exons of the former, encoding a sequence free of mutations. The principal challenge of SMaRT technology is to achieve a reaction as complete as possible, i.e., resulting in 100% repairing of the endogenous mRNA target. The proof of concept of SMaRT feasibility has already been established in several models of genetic diseases caused by recessive mutations. In such cases, in fact, the repair of only a portion of the mutant mRNA pool may be sufficient to obtain a significant therapeutic effect. However in the case of dominant mutations, the target cell must be freed from the majority of mutant mRNA copies, requiring a highly efficient trans?splicing reaction. This likely explains why only a few examples of SMaRT approaches targeting dominant mutations are reported in the literature. In this review, we explain in details the mechanism of trans?splicing, review the different strategies that are under evaluation to lead to efficient trans?splicing, and discuss the advantages and limitations of SMaRT. WIREs RNA 2016, 7:487?498. doi: 10.1002/wrna.1347For further resources related to this article, please visit the WIREs website. ämRNAtrans?splicing in gene therapy for genetic diseases (epmc).pdf DeepDyve Pubget Overpricing