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10.3390/biomedicines6010001 http://scihub22266oqcxt.onion/10.3390/biomedicines6010001 C5874658!5874658!29301272     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       Biomedicines 2018 ; 6 (1): ä Nephropedia Template TP {{tp|p=29301272|t=2018. Skipping Multiple Exons to Treat DMD?Promises and Challenges |pdf=|usr=}}{{29301272}} gab.com Text Skipping Multiple Exons to Treat DMD Promises and Challenges JO: Biomedicines http://www.kidney.de/pget.php?&tw=1&pmid=29301272 #FOAvip Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restoring the reading frame. In 2016, the U.S. Food and Drug Administration (FDA) approved eteplirsen, which skips DMD exon 51 and is applicable to approximately 13% of DMD patients. Multiple exon skipping, which is theoretically applicable to 80?90% of DMD patients in total, have been demonstrated in animal models, including dystrophic mice and dogs, using cocktail antisense oligonucleotides (AOs). Although promising, current drug approval systems pose challenges for the use of a cocktail AO. For example, both exons 6 and 8 need to be skipped to restore the reading frame in dystrophic dogs. Therefore, the cocktail of AOs targeting these exons has a combined therapeutic effect and each AO does not have a therapeutic effect by itself. The current drug approval system is not designed to evaluate such circumstances, which are completely different from cocktail drug approaches in other fields. Significant changes are needed in the drug approval process to promote the cocktail AO approach. Twit Text FOAVip Skipping Multiple Exons to Treat DMD Promises and Challenges ????Biomedicines http://www.kidney.de/pget.php?&tw=1&pmid=29301272 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=29301272 #FOAvip English Wikipedia <ref>{{Cite pmid|29301272}}</ref> Skipping Multiple Exons to Treat DMD?Promises and Challenges #MMPMID29301272 Aslesh T; Maruyama R; Yokota T Biomedicines 2018[Mar]; 6 (1): ä PMID29301272show ga Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restoring the reading frame. In 2016, the U.S. Food and Drug Administration (FDA) approved eteplirsen, which skips DMD exon 51 and is applicable to approximately 13% of DMD patients. Multiple exon skipping, which is theoretically applicable to 80?90% of DMD patients in total, have been demonstrated in animal models, including dystrophic mice and dogs, using cocktail antisense oligonucleotides (AOs). Although promising, current drug approval systems pose challenges for the use of a cocktail AO. For example, both exons 6 and 8 need to be skipped to restore the reading frame in dystrophic dogs. Therefore, the cocktail of AOs targeting these exons has a combined therapeutic effect and each AO does not have a therapeutic effect by itself. The current drug approval system is not designed to evaluate such circumstances, which are completely different from cocktail drug approaches in other fields. Significant changes are needed in the drug approval process to promote the cocktail AO approach. äSkipping Multiple Exons to Treat DMD?Promises and Challenges (epmc).pdf DeepDyve Pubget Overpricing