CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors by Rasmus O. Bak, Matthew H. Porteus

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A look to future directions in gene therapy research for monogenic diseases. by Matthew H Porteus, Jon P Connelly, Shondra M Pruett

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Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus by Kimberly A Wilson, Morgan L Chateau, Matthew H Porteus

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Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels. by Shondra M Pruett-Miller, David W Reading, Shaina N Porter, Matthew H Porteus

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Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor Cells. by Erin H Breese, Corina Buechele, Catherine Dawson, Michael L Cleary, Matthew H Porteus

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Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease by Liwen Xu, Premanjali Lahiri, Jason Skowronski, Neehar Bhatia, Annalisa Lattanzi, Matthew H. Porteus

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Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 by Rasmus O Bak, Daniel P Dever, Andreas Reinisch, David Cruz Hernandez, Ravindra Majeti, Matthew H Porteus

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Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 by Rasmus O Bak, Daniel P Dever, Andreas Reinisch, David Cruz Hernandez, Ravindra Majeti, Matthew H Porteus

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Genome editing in mouse spermatogonial stem/progenitor cells using engineered nucleases. by Danielle A Fanslow, Stacey E Wirt, Jenny C Barker, Jon P Connelly, Matthew H Porteus, Christina Tenenhaus Dann

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Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification by Kimberly A Wilson, Abbye E McEwen, Shondra M Pruett-Miller, Jiuli Zhang, Eric J Kildebeck, Matthew H Porteus

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Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing by Samantha G. Scharenberg, Edina Poletto, Katherine L. Lucot, Pasqualina Colella, Adam Sheikali, Thomas J. Montine, Matthew H. Porteus, Natalia Gomez-Ospina

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Author Correction: Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing by Samantha G. Scharenberg, Edina Poletto, Katherine L. Lucot, Pasqualina Colella, Adam Sheikali, Thomas J. Montine, Matthew H. Porteus, Natalia Gomez-Ospina

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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice by Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus

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Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell trans... by Volker Wiebking, Ciaran M. Lee, Nathalie Mostrel, Premanjali Lahiri, Rasmus Bak, Gang Bao, Maria Grazia Roncarolo, Alice Bertaina, Matthew H. Porteus

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Viral single-strand DNA induces p53-dependent apoptosis in human embryonic stem cells. by Matthew L Hirsch, B Matthew Fagan, Raluca Dumitru, Jacquelyn J Bower, Swati Yadav, Matthew H Porteus, Larysa H Pevny, R Jude Samulski

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Investigation of Cas9 antibodies in the human eye by Marcus A. Toral, Carsten T. Charlesworth, Benjamin Ng, Teja Chemudupati, Shota Homma, Hiromitsu Nakauchi, Alexander G. Bassuk, Matthew H. Porteus, Vinit B. Mahajan

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Author Correction: Investigation of Cas9 antibodies in the human eye by Marcus A. Toral, Carsten T. Charlesworth, Benjamin Ng, Teja Chemudupati, Shota Homma, Hiromitsu Nakauchi, Alexander G. Bassuk, Matthew H. Porteus, Vinit B. Mahajan

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The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells by Rajiv Sharma, Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena, Thomas Köhnke, Gang Bao, Matthew H. Porteus, Ravindra Majeti

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Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting by Carsten T. Charlesworth, Joab Camarena, M. Kyle Cromer, Sriram Vaidyanathan, Rasmus O. Bak, Jason M. Carte, Jason Potter, Daniel P. Dever, Matthew H. Porteus

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Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards by Renata M. Martin, Jonas L. Fowler, M. Kyle Cromer, Benjamin J. Lesch, Ezequiel Ponce, Nobuko Uchida, Toshinobu Nishimura, Matthew H. Porteus, Kyle M. Loh

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