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10.1007/s00439-016-1725-z http://scihub22266oqcxt.onion/10.1007/s00439-016-1725-z C5006996!5006996!27542949     free     free     free Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Hum+Genet 2016 ; 135 (9): 1029-40 Nephropedia Template TP {{tp|p=27542949|t=2016. Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy |pdf=|usr=}}{{27542949}} gab.com Text Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy JO: Hum Genet http://www.kidney.de/pget.php?&tw=1&pmid=27542949 #FOAvip Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is caused by mutations to the DMD gene that encodes the dystrophin protein. Recent advances in genome editing and gene therapy offer hope for the development of potential therapeutics. Truncated versions of the DMD gene can be delivered to the affected tissues with viral vectors and show promising results in a variety of animal models. Genome editing with the CRISPR/Cas9 system has recently been used to restore dystrophin expression by deleting one or more exons of the DMD gene in patient cells and in a mouse model that led to functional improvement of muscle strength. Exon skipping with oligonucleotides has been successful in several animal models and evaluated in multiple clinical trials. Next-generation oligonucleotide formulations offer significant promise to build on these results. All these approaches to restoring dystrophin expression are encouraging, but many hurdles remain. This review summarizes the current state of these technologies and summarizes considerations for their future development. Twit Text FOAVip Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy ????Hum Genet http://www.kidney.de/pget.php?&tw=1&pmid=27542949 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27542949 #FOAvip English Wikipedia <ref>{{Cite pmid|27542949}}</ref> Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy #MMPMID27542949 Robinson-Hamm JN; Gersbach CA Hum Genet 2016[Sep]; 135 (9): 1029-40 PMID27542949show ga Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is caused by mutations to the DMD gene that encodes the dystrophin protein. Recent advances in genome editing and gene therapy offer hope for the development of potential therapeutics. Truncated versions of the DMD gene can be delivered to the affected tissues with viral vectors and show promising results in a variety of animal models. Genome editing with the CRISPR/Cas9 system has recently been used to restore dystrophin expression by deleting one or more exons of the DMD gene in patient cells and in a mouse model that led to functional improvement of muscle strength. Exon skipping with oligonucleotides has been successful in several animal models and evaluated in multiple clinical trials. Next-generation oligonucleotide formulations offer significant promise to build on these results. All these approaches to restoring dystrophin expression are encouraging, but many hurdles remain. This review summarizes the current state of these technologies and summarizes considerations for their future development. äGene therapies that restore dystrophin expression for the treatment of(epmc).pdf DeepDyve Pubget Overpricing