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10.1016/j.cell.2017.07.010 http://scihub22266oqcxt.onion/10.1016/j.cell.2017.07.010 C5873302!5873302!28803727     free     free     free Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 225.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Cell 2017 ; 170 (5): 899-912.e10 Nephropedia Template TP {{tp|p=28803727|t=2017. Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9 |pdf=|usr=}}{{28803727}} gab.com Text Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9 JO: Cell http://www.kidney.de/pget.php?&tw=1&pmid=28803727 #FOAvip Microsatellite repeat expansions in DNA produce pathogenic RNA species that cause dominantly inherited diseases such as myotonic dystrophy type 1 and 2 (DM1/2), Huntington?s disease, and C9orf72-linked amyotrophic lateral sclerosis (C9-ALS). Means to target these repetitive RNAs are required for diagnostic and therapeutic purposes. Here, we describe the development of a programmable CRISPR system capable of specifically visualizing and eliminating these toxic RNAs. We observe specific targeting and efficient elimination of micro-satellite repeat expansion RNAs both when exogenously expressed and in patient cells. Importantly, RNA-targeting Cas9 (RCas9) reverses hallmark features of disease including elimination of RNA foci among all conditions studied (DM1, DM2, C9-ALS, polyglutamine diseases), reduction of polyglutamine protein products, relocalization of repeat-bound proteins to resemble healthy controls, and efficient reversal of DM1-associated splicing abnormalities in patient myotubes. Finally, we report a truncated RCas9 system compatible with adeno-associated viral packaging. This effort highlights the potential of RCas9 for human therapeutics. Twit Text FOAVip Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9 ????Cell http://www.kidney.de/pget.php?&tw=1&pmid=28803727 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=28803727 #FOAvip English Wikipedia <ref>{{Cite pmid|28803727}}</ref> Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9 #MMPMID28803727 Batra R; Nelles DA; Pirie E; Blue SM; Marina RJ; Wang H; Chaim IA; Thomas JD; Zhang N; Nguyen V; Aigner S; Markmiller S; Xia G; Corbett KD; Swanson MS; Yeo GW Cell 2017[Aug]; 170 (5): 899-912.e10 PMID28803727show ga Microsatellite repeat expansions in DNA produce pathogenic RNA species that cause dominantly inherited diseases such as myotonic dystrophy type 1 and 2 (DM1/2), Huntington?s disease, and C9orf72-linked amyotrophic lateral sclerosis (C9-ALS). Means to target these repetitive RNAs are required for diagnostic and therapeutic purposes. Here, we describe the development of a programmable CRISPR system capable of specifically visualizing and eliminating these toxic RNAs. We observe specific targeting and efficient elimination of micro-satellite repeat expansion RNAs both when exogenously expressed and in patient cells. Importantly, RNA-targeting Cas9 (RCas9) reverses hallmark features of disease including elimination of RNA foci among all conditions studied (DM1, DM2, C9-ALS, polyglutamine diseases), reduction of polyglutamine protein products, relocalization of repeat-bound proteins to resemble healthy controls, and efficient reversal of DM1-associated splicing abnormalities in patient myotubes. Finally, we report a truncated RCas9 system compatible with adeno-associated viral packaging. This effort highlights the potential of RCas9 for human therapeutics. äElimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Target(epmc).pdf DeepDyve Pubget Overpricing