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10.1016/j.ebiom.2018.02.015 http://scihub22266oqcxt.onion/10.1016/j.ebiom.2018.02.015 C5925576!5925576 !29500128     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 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 267.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 267.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 267.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\29500128 .jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117       EBioMedicine 2018 ; 29 (ä): 104-111 Nephropedia Template TP {{tp|p=29500128 |t=2018. Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates ?1-antitrypsin Deficiency Phenotype |pdf=|usr=}}{{29500128 }} gab.com Text Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates 1-antitrypsin Deficiency Phenotype JO: EBioMedicine http://www.kidney.de/pget.php?&tw=1&pmid=29500128 #FOAvip ?1-antitrypsin (AAT) is a circulating serine protease inhibitor secreted from the liver and important in preventing proteolytic neutrophil elastase associated tissue damage, primarily in lungs. In humans, AAT is encoded by the SERPINA1 (hSERPINA1) gene in which a point mutation (commonly referred to as PiZ) causes aggregation of the miss-folded protein in hepatocytes resulting in subsequent liver damage. In an attempt to rescue the pathologic liver phenotype of a mouse model of human AAT deficiency (AATD), we used adenovirus to deliver Cas9 and a guide-RNA (gRNA) molecule targeting hSERPINA1. Our single dose therapeutic gene editing approach completely reverted the phenotype associated with the PiZ mutation, including circulating transaminase and human AAT (hAAT) protein levels, liver fibrosis and protein aggregation. Furthermore, liver histology was significantly improved regarding inflammation and overall morphology in hSERPINA1 gene edited PiZ mice. Genomic analysis confirmed significant disruption to the hSERPINA1 transgene resulting in a reduction of hAAT protein levels and quantitative mRNA analysis showed a reduction in fibrosis and hepatocyte proliferation as a result of editing. Our findings indicate that therapeutic gene editing in hepatocytes is possible in an AATD mouse model. Twit Text FOAVip Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates 1-antitrypsin Deficiency Phenotype ????EBioMedicine http://www.kidney.de/pget.php?&tw=1&pmid=29500128 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=29500128 #FOAvip English Wikipedia <ref>{{Cite pmid|29500128 }}</ref> Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates ?1-antitrypsin Deficiency Phenotype #MMPMID29500128 Bjursell M ; Porritt MJ ; Ericson E ; Taheri-Ghahfarokhi A ; Clausen M ; Magnusson L ; Admyre T ; Nitsch R ; Mayr L ; Aasehaug L ; Seeliger F ; Maresca M ; Bohlooly-Y M ; Wiseman J EBioMedicine 2018[Mar]; 29 (ä): 104-111 PMID29500128 show ga ?1-antitrypsin (AAT) is a circulating serine protease inhibitor secreted from the liver and important in preventing proteolytic neutrophil elastase associated tissue damage, primarily in lungs. In humans, AAT is encoded by the SERPINA1 (hSERPINA1) gene in which a point mutation (commonly referred to as PiZ) causes aggregation of the miss-folded protein in hepatocytes resulting in subsequent liver damage. In an attempt to rescue the pathologic liver phenotype of a mouse model of human AAT deficiency (AATD), we used adenovirus to deliver Cas9 and a guide-RNA (gRNA) molecule targeting hSERPINA1. Our single dose therapeutic gene editing approach completely reverted the phenotype associated with the PiZ mutation, including circulating transaminase and human AAT (hAAT) protein levels, liver fibrosis and protein aggregation. Furthermore, liver histology was significantly improved regarding inflammation and overall morphology in hSERPINA1 gene edited PiZ mice. Genomic analysis confirmed significant disruption to the hSERPINA1 transgene resulting in a reduction of hAAT protein levels and quantitative mRNA analysis showed a reduction in fibrosis and hepatocyte proliferation as a result of editing. Our findings indicate that therapeutic gene editing in hepatocytes is possible in an AATD mouse model. |*CRISPR-Cas Systems [MESH] |*Gene Editing [MESH] |*Phenotype [MESH] |Adenoviridae/genetics [MESH] |Animals [MESH] |Cell Proliferation [MESH] |Disease Models, Animal [MESH] |Gene Expression [MESH] |Genetic Vectors/genetics [MESH] |Humans [MESH] |Mice [MESH] |Mice, Transgenic [MESH] |Transduction, Genetic [MESH] |Transgenes [MESH] |alpha 1-Antitrypsin Deficiency/*genetics/*metabolism/pathology/therapy [MESH]Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model(epmc).pdf DeepDyve Pubget Overpricing