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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

Quantifying Genome-Editing Outcomes at Endogenous Loci with SMRT Sequencing by Ayal Hendel, Eric J. Kildebeck, Eli J. Fine, Joseph T. Clark, Niraj Punjya, Vittorio Sebastiano, Gang Bao, Matthew H. Porteus

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

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

Get full text

DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis by Jennifer E. Adair, Mark R. Enstrom, Kevin G. Haworth, Lauren E. Schefter, Reza Shahbazi, Daniel R. Humphrys, Shaina Porter, Kenric Tam, Matthew H. Porteus, Hans-Peter Kiem

Get full text

Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells by M. Kyle Cromer, Valentin V. Barsan, Erich Jaeger, Mengchi Wang, Jessica P. Hampton, Feng Chen, Drew Kennedy, Jenny Xiao, Irina Khrebtukova, Ana Granat, Tiffany Truong, Matthew H. Porteus

Get full text

Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation by Francisco X Galdos, Carissa Lee, Soah Lee, Sharon Paige, William Goodyer, Sidra Xu, Tahmina Samad, Gabriela V Escobar, Adrija Darsha, Aimee Beck, Rasmus O Bak, Matthew H Porteus, Sean M Wu

Get full text