CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

by Rasmus O. Bak, Matthew H. Porteus
Published 2017-07-01

A look to future directions in gene therapy research for monogenic diseases.

by Matthew H Porteus, Jon P Connelly, Shondra M Pruett
Published 2006-09-01

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
Published 2013-01-01

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
Published 2015-01-01

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
Published 2009-02-01

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
Published 2018-11-01

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
Published 2017-09-01

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
Published 2014-01-01

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
Published 2013-01-01

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
Published 2014-04-01

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
Published 2020-07-01

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
Published 2021-01-01

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
Published 2020-08-01

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
Published 2011-01-01

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
Published 2021-01-01

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
Published 2018-09-01

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
Published 2020-06-01

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
Published 2020-06-01

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

by Sriram Vaidyanathan, Krist T. Azizian, A.K.M. Ashiqul Haque, Jordana M. Henderson, Ayal Hendel, Sabrina Shore, Justin S. Antony, Richard I. Hogrefe, Michael S.D. Kormann, Matthew H. Porteus, Anton P. McCaffrey
Published 2018-09-01

Functional significance of U2AF1 S34F mutations in lung adenocarcinomas

by Mohammad S. Esfahani, Luke J. Lee, Young-Jun Jeon, Ryan A. Flynn, Henning Stehr, Angela B. Hui, Noriko Ishisoko, Eric Kildebeck, Aaron M. Newman, Scott V. Bratman, Matthew H. Porteus, Howard Y. Chang, Ash A. Alizadeh, Maximilian Diehn
Published 2019-12-01