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10.3390/ijms17101609 http://scihub22266oqcxt.onion/10.3390/ijms17101609 C5085642!5085642!27669223     free     free     free Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Int+J+Mol+Sci 2016 ; 17 (10): ä Nephropedia Template TP {{tp|p=27669223|t=2016. Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair |pdf=|usr=}}{{27669223}} gab.com Text Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair JO: Int J Mol Sci http://www.kidney.de/pget.php?&tw=1&pmid=27669223 #FOAvip RNA trans-splicing is a promising tool for mRNA modification in a diversity of genetic disorders. In particular, the substitution of internal exons of a gene by combining 3? and 5? RNA trans-splicing seems to be an elegant way to modify especially large pre-mRNAs. Here we discuss a robust method for designing double RNA trans-splicing molecules (dRTM). We demonstrate how the technique can be implemented in an endogenous setting, using COL7A1, the gene encoding type VII collagen, as a target. An RTM screening system was developed with the aim of testing the replacement of two internal COL7A1 exons, harbouring a homozygous mutation, with the wild-type version. The most efficient RTMs from a pool of randomly generated variants were selected via our fluorescence-based screening system and adapted for use in an in vitro disease model system. Transduction of type VII collagen-deficient keratinocytes with the selected dRTM led to accurate replacement of two internal COL7A1 exons resulting in a restored wild-type RNA sequence. This is the first study demonstrating specific exon replacement by double RNA trans-splicing within an endogenous transcript in cultured cells, corroborating the utility of this technology for mRNA repair in a variety of genetic disorders. Twit Text FOAVip Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair ????Int J Mol Sci http://www.kidney.de/pget.php?&tw=1&pmid=27669223 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27669223 #FOAvip English Wikipedia <ref>{{Cite pmid|27669223}}</ref> Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair #MMPMID27669223 Hüttner C; Murauer EM; Hainzl S; Kocher T; Neumayer A; Reichelt J; Bauer JW; Koller U Int J Mol Sci 2016[Oct]; 17 (10): ä PMID27669223show ga RNA trans-splicing is a promising tool for mRNA modification in a diversity of genetic disorders. In particular, the substitution of internal exons of a gene by combining 3? and 5? RNA trans-splicing seems to be an elegant way to modify especially large pre-mRNAs. Here we discuss a robust method for designing double RNA trans-splicing molecules (dRTM). We demonstrate how the technique can be implemented in an endogenous setting, using COL7A1, the gene encoding type VII collagen, as a target. An RTM screening system was developed with the aim of testing the replacement of two internal COL7A1 exons, harbouring a homozygous mutation, with the wild-type version. The most efficient RTMs from a pool of randomly generated variants were selected via our fluorescence-based screening system and adapted for use in an in vitro disease model system. Transduction of type VII collagen-deficient keratinocytes with the selected dRTM led to accurate replacement of two internal COL7A1 exons resulting in a restored wild-type RNA sequence. This is the first study demonstrating specific exon replacement by double RNA trans-splicing within an endogenous transcript in cultured cells, corroborating the utility of this technology for mRNA repair in a variety of genetic disorders. äDesigning Efficient Double RNA trans-Splicing Molecules for Targeted R(epmc).pdf DeepDyve Pubget Overpricing