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Matthew Porteus, MD, PhD
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Journal Articles

Adenylate Kinase 2 Links Energy Metabolism and Cell Fate in Hematopoietic Stem and Progenitor Cells

Wenqing Wang, PhD, Avni Awani, PhD, Andrew Devilbiss, PhD, Thomas Mathews, PhD, Daniel Thomas, MD PhD, Daniel P. Dever, PhD, Matthew Porteus, MD PhD, Sean Morrison, PhD, Katja G Weinacht, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3705.
https://doi.org/10.1182/blood-2019-129424
Published: 2019
Journal Articles

An Engineered Cell-Traceable Model of Reticular Dysgenesis in Human Hematopoietic Stem Cells Linking Metabolism and Differentiation

Wenqing Wang, PhD, Avni Awani, PhD, Lauren Reich, Yusuke Nakauchi, MD PhD, Daniel Thomas, MD PhD, Daniel P. Dever, PhD, Matthew Porteus, MD PhD, Katja G. Weinacht, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2558.
https://doi.org/10.1182/blood-2018-99-117926
Published: 2018
Journal Articles

Pharmacological Inhibition of Nlk (Nemo-like Kinase) Rescues Erythropoietic Defects in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, Jun Chen, MD PhD, Kavitha Siva, PhD, Gianluca Veretti, PhD, Daniel P. Dever, PhD, Minyoung Youn, PhD, Hee-Don Chae, PhD, Jacqueline D Mercado, Mallika Saxena, Anupama Narla, MD, Bertil Glader, MD, Matthew Porteus, MD PhD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 754.
https://doi.org/10.1182/blood-2018-99-119954
Published: 2018
Journal Articles

Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency

Oscar Quintana Bustamante, PhD, Sara Fañanas-Baquero, Daniel P. Dever, PhD, Alberquilla Omaira, Joab Camarena, BS, Rebeca Sanchez-Dominguez, PhD, Matias Morin, Val Fernandez, Miguel Angel Moreno-Pelayo, Juan A. Bueren, PhD, Matthew Porteus, MD PhD, Jose C Segovia, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 5792.
https://doi.org/10.1182/blood-2018-99-111772
Published: 2018
Journal Articles

Highly Efficient Editing of the Beta-Globin Gene in Patient Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease

So Hyun Park, BS, Ciaran M Lee, PhD, Daniel P. Dever, PhD, Timothy H Davis, BS, Joab Camarena, BS, Yankai Zhang, PhD, Matthew Porteus, MD PhD, Vivian A Sheehan, MD PhD, Gang Bao, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2192.
https://doi.org/10.1182/blood-2018-99-117371
Published: 2018
Journal Articles

CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.

Michelle I. Lin, PhD, Elizabeth Paik, PhD, Bibhu Mishra, PhD, David Burkhardt, Andrew Kernytsky, PhD, Michael Pettiglio, Yi-Shan Chen, Kaleigh Tomkinson, Amanda Woo, Mauricio Cortes, PhD, Siyuan Tan, PhD, Todd D Borland, BS, Lawrence Klein, PhD, Angela Yen, PhD, Sudipta Mahajan, Eric Chan, PhD, Brenda Eustace, PhD, Matthew Porteus, MD PhD, Tirtha Chakraborty, PhD, Chad Cowan, Rodger Novak, MD, Ante Lundberg, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 284.
https://doi.org/10.1182/blood.V130.Suppl_1.284.284
Published: 2017
Journal Articles

Preclinical Development of HBB Gene Correction in Autologous Hematopoietic Stem and Progenitor Cells to Treat Severe Sickle Cell Disease

Daniel P. Dever, PhD, Joab Camarena, BS, Ciaran Lee, PhD, Christopher Vakulskas, PhD, Mark Behlke, MD PhD, Gang Bao, PhD, Matthew Porteus, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4620.
https://doi.org/10.1182/blood.V130.Suppl_1.4620.4620
Published: 2017
Journal Articles

Priming Human Hematopoietic Stem and Progenitor Cells for Cas9/Sgrna and rAAV6-Mediated Homologous Recombination

Joab Camarena, BS, Carsten T Charlesworth, BS, Michael Kyle Cromer, BS, MPhil, PhD, Sriram Vaidyanathan, PhD, Rasmus O Bak, PhD, Jason Carte, PhD, Jason Potter, PhD, Daniel P. Dever, PhD, Matthew Porteus, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3342.
https://doi.org/10.1182/blood.V130.Suppl_1.3342.3342
Published: 2017
Journal Articles

Dual-Method Clone Tracking in Nonhuman Primates Confirms Long-Term Hematopoietic Reconstitution Initiated By Early Engrafting Clones

Jennifer E Adair, PhD, Zachary K. Norgaard, MSc, Kevin G Haworth, PhD, Lauren E Schefter, BA, Kenric Tam, MD, Matthew Porteus, MD PhD, Hans-Peter Kiem, MD FACP
Journal: Blood
Blood (2016) 128 (22): 1475.
https://doi.org/10.1182/blood.V128.22.1475.1475
Published: 2016
Journal Articles

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Chiara Antoniani, MSc, Vasco Meneghini, Annalisa Lattanzi, PhD, Giulia Pavani, PhD, Tristan Felix, MSc, Fatima Amor, MSc, Oriana Romano, Elisa Magrin, PhD, Leslie Weber, MSc, Thomas Cradick, Ante S Lundberg, MD, Matthew Porteus, MD PhD, Mario Amendola, Marina Cavazzana, MD PhD, Fulvio Mavilio, PhD, Annarita Miccio, PhD
Journal: Blood
Blood (2016) 128 (22): 321.
https://doi.org/10.1182/blood.V128.22.321.321
Published: 2016
Journal Articles

Beta-Globin Gene Targeting in Human Hematopoietic Stem Cells Using Cas9 Ribonucleoprotein and rAAV6

Daniel Patrick Dever, PhD, Matthew Porteus, MD PhD
Journal: Blood
Blood (2016) 128 (22): 2310.
https://doi.org/10.1182/blood.V128.22.2310.2310
Published: 2016
Journal Articles

Anti-Fungal Prophylaxis Using Intermediate Dose Ambisome Is Associated with Delayed Methotrexate Clearance in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Liora M. Schultz, MD, Karan Kumar, MD, Suzette Stone, Elizabeth Callard, Jessica Witkowski, MD, Leigh Shinn, Lisa Pinner, Shizuka Franklin, Julianna Kula, Namrata Patel, Prianka Kumar, Kenneth I. Weinberg, MD, Matthew Porteus, MD PhD, Rajni Agarwal, MD, Ami Shah, MD, Sandhya Kharbanda, MD
Journal: Blood
Blood (2016) 128 (22): 5762.
https://doi.org/10.1182/blood.V128.22.5762.5762
Published: 2016
Journal Articles

Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia

Michelle I Lin, PhD, Elizabeth J Paik, PhD, Bibhu P Mishra, PhD, Song Chou, PhD, Ying Zhang, PhD, Kaleigh Tomkinson, MS, Michael A Pettiglio, BS, Andrew Sanginario, BS, Amanda Woo, BS, Min Jin Lee, BS, Melanie Allen, MS, Thomas J Cradick, PhD, Tirtha Chakraborty, PhD, Matthew Porteus, MD PhD, Ciaran M Lee, PhD, Gang Bao, PhD, Annarita Miccio, PhD, Annalisa Lattanzi, PhD, Fulvio Mavilio, PhD, Chad A Cowan, PhD, Rodger Novak, MD, Ante S Lundberg, MD
Journal: Blood
Blood (2016) 128 (22): 4708.
https://doi.org/10.1182/blood.V128.22.4708.4708
Published: 2016
Journal Articles

Illuminating Clonal Dynamics of a Leukemia Cell Line Using Viral Barcoding and Illumina Deep Sequencing Technology

Shaina Porter, BS, Gareth Highnam, BS, David Mittelman, PhD, Matthew Porteus, MD, PhD
Journal: Blood
Blood (2012) 120 (21): 1410.
https://doi.org/10.1182/blood.V120.21.1410.1410
Published: 2012
Journal Articles

A Biological Delivery Platform for Zinc Finger Nucleases Using Transferrin-Mediated Endocytosis

Steffen E. Meiler, MD, William S. Dynan, PhD, Matthew Porteus, MD, PhD, David L. Spector, PhD, Gang Bao, PhD, Zhong Chen, MD, Jay Pandey, PhD, Deepika Goyal, PhD, R. Ileng Kumaran, PhD, Marlene Wade, Davies Agyekum
Journal: Blood
Blood (2011) 118 (21): 1071.
https://doi.org/10.1182/blood.V118.21.1071.1071
Published: 2011