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10.1007/s00439-016-1699-x http://scihub22266oqcxt.onion/10.1007/s00439-016-1699-x C4999461!4999461!27357631     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=27357631&cmd=llinks): Failed to open stream: HTTP request failed! HTTP/1.1 429 Too Many Requests in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 215 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Hum+Genet 2016 ; 135 (9): 977-81 Nephropedia Template TP {{tp|p=27357631|t=2016. Genome-editing technologies for gene correction of hemophilia |pdf=|usr=}}{{27357631}} gab.com Text Genome-editing technologies for gene correction of hemophilia JO: Hum Genet http://www.kidney.de/pget.php?&tw=1&pmid=27357631 #FOAvip Hemophilia is caused by various mutations in blood coagulation factor genes, including factor VIII (FVIII) and factor IX (FIX), that encode key proteins in the blood clotting pathway. Although the addition of therapeutic genes or infusion of clotting factors may be used to remedy hemophilia?s symptoms, no permanent cure for the disease exists. Moreover, patients often develop neutralizing antibodies or experience adverse effects that limit the therapy?s benefits. However, targeted gene therapy involving the precise correction of these mutated genes at the genome level using programmable nucleases is a promising strategy. These nucleases can induce double-strand breaks (DSBs) on genomes, and repairs of such induced DSBs by the two cellular repair systems enable a targeted gene correction. Going beyond cultured cell systems, we are now entering the age of direct gene correction in vivo using various delivery tools. Here, we describe the current status of in vivo and ex vivo genome-editing technology related to potential hemophilia gene correction and the prominent issues surrounding its application in patients with monogenic diseases. Twit Text FOAVip Genome-editing technologies for gene correction of hemophilia ????Hum Genet http://www.kidney.de/pget.php?&tw=1&pmid=27357631 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27357631 #FOAvip English Wikipedia <ref>{{Cite pmid|27357631}}</ref> Genome-editing technologies for gene correction of hemophilia #MMPMID27357631 Park CY; Lee DR; Sung JJ; Kim DW Hum Genet 2016[]; 135 (9): 977-81 PMID27357631show ga Hemophilia is caused by various mutations in blood coagulation factor genes, including factor VIII (FVIII) and factor IX (FIX), that encode key proteins in the blood clotting pathway. Although the addition of therapeutic genes or infusion of clotting factors may be used to remedy hemophilia?s symptoms, no permanent cure for the disease exists. Moreover, patients often develop neutralizing antibodies or experience adverse effects that limit the therapy?s benefits. However, targeted gene therapy involving the precise correction of these mutated genes at the genome level using programmable nucleases is a promising strategy. These nucleases can induce double-strand breaks (DSBs) on genomes, and repairs of such induced DSBs by the two cellular repair systems enable a targeted gene correction. Going beyond cultured cell systems, we are now entering the age of direct gene correction in vivo using various delivery tools. Here, we describe the current status of in vivo and ex vivo genome-editing technology related to potential hemophilia gene correction and the prominent issues surrounding its application in patients with monogenic diseases. äGenome-editing technologies for gene correction of hemophilia (epmc).pdf DeepDyve Pubget Overpricing