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10.1038/srep12065 http://scihub22266oqcxt.onion/10.1038/srep12065 C4496796!4496796!26156589     free     free     free Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 219.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 253.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Sci+Rep 2015 ; 5 (ä): ä Nephropedia Template TP {{tp|p=26156589|t=2015. Both TALENs and CRISPR/Cas9 directly target the HBB IVS2?654 (C? ?T) mutation in ?-thalassemia-derived iPSCs |pdf=|usr=}}{{26156589}} gab.com Text Both TALENs and CRISPR/Cas9 directly target the HBB IVS2 654 (C T) mutation in -thalassemia-derived iPSCs JO: Sci Rep http://www.kidney.de/pget.php?&tw=1&pmid=26156589 #FOAvip ?-Thalassemia is one of the most common genetic blood diseases and is caused by either point mutations or deletions in the ?-globin (HBB) gene. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations may be a potential therapeutic strategy for this disease. Due to the low efficiency of typical homologous recombination, endonucleases, including TALENs and CRISPR/Cas9, have been widely used to enhance the gene correction efficiency in patient-derived iPSCs. Here, we designed TALENs and CRISPR/Cas9 to directly target the intron2 mutation site IVS2-654 in the globin gene. We observed different frequencies of double-strand breaks (DSBs) at IVS2-654 loci using TALENs and CRISPR/Cas9, and TALENs mediated a higher homologous gene targeting efficiency compared to CRISPR/Cas9 when combined with the piggyBac transposon donor. In addition, more obvious off-target events were observed for CRISPR/Cas9 compared to TALENs. Finally, TALENs-corrected iPSC clones were selected for erythroblast differentiation using the OP9 co-culture system and detected relatively higher transcription of HBB than the uncorrected cells. This comparison of using TALENs or CRISPR/Cas9 to correct specific HBB mutations in patient-derived iPSCs will guide future applications of TALENs- or CRISPR/Cas9-based gene therapies in monogenic diseases. Twit Text FOAVip Both TALENs and CRISPR/Cas9 directly target the HBB IVS2 654 (C T) mutation in -thalassemia-derived iPSCs ????Sci Rep http://www.kidney.de/pget.php?&tw=1&pmid=26156589 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=26156589 #FOAvip English Wikipedia <ref>{{Cite pmid|26156589}}</ref> Both TALENs and CRISPR/Cas9 directly target the HBB IVS2?654 (C? ?T) mutation in ?-thalassemia-derived iPSCs #MMPMID26156589 Xu P; Tong Y; Liu Xz; Wang Tt; Cheng L; Wang By; Lv X; Huang Y; Liu Dp Sci Rep 2015[]; 5 (ä): ä PMID26156589show ga ?-Thalassemia is one of the most common genetic blood diseases and is caused by either point mutations or deletions in the ?-globin (HBB) gene. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations may be a potential therapeutic strategy for this disease. Due to the low efficiency of typical homologous recombination, endonucleases, including TALENs and CRISPR/Cas9, have been widely used to enhance the gene correction efficiency in patient-derived iPSCs. Here, we designed TALENs and CRISPR/Cas9 to directly target the intron2 mutation site IVS2-654 in the globin gene. We observed different frequencies of double-strand breaks (DSBs) at IVS2-654 loci using TALENs and CRISPR/Cas9, and TALENs mediated a higher homologous gene targeting efficiency compared to CRISPR/Cas9 when combined with the piggyBac transposon donor. In addition, more obvious off-target events were observed for CRISPR/Cas9 compared to TALENs. Finally, TALENs-corrected iPSC clones were selected for erythroblast differentiation using the OP9 co-culture system and detected relatively higher transcription of HBB than the uncorrected cells. This comparison of using TALENs or CRISPR/Cas9 to correct specific HBB mutations in patient-derived iPSCs will guide future applications of TALENs- or CRISPR/Cas9-based gene therapies in monogenic diseases. äBoth TALENs and CRISPR/Cas9 directly target the HBB IVS2?654 (C? ?T) m(epmc).pdf DeepDyve Pubget Overpricing