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10.1038/mt.2016.148 http://scihub22266oqcxt.onion/10.1038/mt.2016.148 C5113113!5113113!27406980     free     free     free Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 233.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Mol+Ther 2016 ; 24 (9): 1561-9 Nephropedia Template TP {{tp|p=27406980|t=2016. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells |pdf=|usr=}}{{27406980}} gab.com Text CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells JO: Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=27406980 #FOAvip Targeted genome editing technology can correct the sickle cell disease mutation of the ?-globin gene in hematopoietic stem cells. This correction supports production of red blood cells that synthesize normal hemoglobin proteins. Here, we demonstrate that Transcription Activator-Like Effector Nucleases (TALENs) and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system can target DNA sequences around the sickle-cell mutation in the ?-globin gene for site-specific cleavage and facilitate precise correction when a homologous donor template is codelivered. Several pairs of TALENs and multiple CRISPR guide RNAs were evaluated for both on-target and off-target cleavage rates. Delivery of the CRISPR/Cas9 components to CD34+ cells led to over 18% gene modification in vitro. Additionally, we demonstrate the correction of the sickle cell disease mutation in bone marrow derived CD34+ hematopoietic stem and progenitor cells from sickle cell disease patients, leading to the production of wild-type hemoglobin. These results demonstrate correction of the sickle mutation in patient-derived CD34+ cells using CRISPR/Cas9 technology. Twit Text FOAVip CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells ????Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=27406980 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27406980 #FOAvip English Wikipedia <ref>{{Cite pmid|27406980}}</ref> CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells #MMPMID27406980 Hoban MD; Lumaquin D; Kuo CY; Romero Z; Long J; Ho M; Young CS; Mojadidi M; Fitz-Gibbon S; Cooper AR; Lill GR; Urbinati F; Campo-Fernandez B; Bjurstrom CF; Pellegrini M; Hollis RP; Kohn DB Mol Ther 2016[Sep]; 24 (9): 1561-9 PMID27406980show ga Targeted genome editing technology can correct the sickle cell disease mutation of the ?-globin gene in hematopoietic stem cells. This correction supports production of red blood cells that synthesize normal hemoglobin proteins. Here, we demonstrate that Transcription Activator-Like Effector Nucleases (TALENs) and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system can target DNA sequences around the sickle-cell mutation in the ?-globin gene for site-specific cleavage and facilitate precise correction when a homologous donor template is codelivered. Several pairs of TALENs and multiple CRISPR guide RNAs were evaluated for both on-target and off-target cleavage rates. Delivery of the CRISPR/Cas9 components to CD34+ cells led to over 18% gene modification in vitro. Additionally, we demonstrate the correction of the sickle cell disease mutation in bone marrow derived CD34+ hematopoietic stem and progenitor cells from sickle cell disease patients, leading to the production of wild-type hemoglobin. These results demonstrate correction of the sickle mutation in patient-derived CD34+ cells using CRISPR/Cas9 technology. äCRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ (epmc).pdf DeepDyve Pubget Overpricing