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Matthew H Porteus
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Journal Articles

Targeting the JAK2- V617F Mutation in Polycythemia Vera Using CRISPR/AAV6 Genome Editing

Freja Ekman, Sridhar Selvaraj, Jason Gotlib, M. Kyle Cromer, Matthew H. Porteus
Journal: Blood
Blood (2024) 144 (Supplement 1): 4524.
https://doi.org/10.1182/blood-2024-211120
Published: 2024
Journal Articles

Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesis

Wenqing Wang, Martin Arreola, Thomas Mathews, Andrew W DeVilbiss, Zhiyu Zhao, Misty Martin-Sandoval, Abdulvasey Mohammed, Giorgia Benegiamo, Avni Awani, Ludger J. E. Goeminne, Daniel Patrick Dever, Yusuke Nakauchi, Matthew H Porteus, Mara Pavel-Dinu, Waleed Al Herz, Johan Auwerx, Sean J. Morrison, Katja G. Weinacht
Journal: Blood
Blood blood.2024024123.
https://doi.org/10.1182/blood.2024024123
Published: 2024
Includes: Supplemental data
Journal Articles

Engineered Single Amino Acid Substitutions Protect Hematopoietic Stem and Progenitor Cells from CD123 Targeted Immunotherapy

Emmanuelle Landmann, Anna Devaux, Rosalba Lepore, Romina Marone, Corinne Engdahl, Marko Hasiuk, Giuseppina Capoferri, Amélie Wiederkehr, Lisa C Wellinger, Alessandro Sinopoli, Valentin Do Sacramento, Anna Haydn, Laura Garcia Prat, Christopher Divsalar, Anna Camus, Julie Brault, Liwen Xu, Torsten Schwede, Matthew H. Porteus, Stefanie Urlinger, Lukas T Jeker
Journal: Blood
Blood (2022) 140 (Supplement 1): 5724–5725.
https://doi.org/10.1182/blood-2022-163815
Published: 2022
Journal Articles

Novel Humanized Loss-of-Function NF1 Mouse Model of Juvenile Myelomonocytic Leukemia

Roshani Sinha, Rachana Patil, Rosa Romano, Esmond Lee, Zhenyu Yao, Y. Lucy Liu, Matthew H. Porteus, Alice Bertaina
Journal: Blood
Blood (2022) 140 (Supplement 1): 9765–9766.
https://doi.org/10.1182/blood-2022-167413
Published: 2022
Journal Articles

Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD

Julie Kanter, John F. DiPersio, Patrick Leavey, David C. Shyr, Alexis A Thompson, Matthew H. Porteus, Allison Intondi, Premanjali Lahiri, Daniel P. Dever, Alexandria Petrusich, Joshua Lehrer-Graiwer
Journal: Blood
Blood (2021) 138 (Supplement 1): 1864.
https://doi.org/10.1182/blood-2021-152892
Published: 2021
Journal Articles

Reticular Dysgenesis-Associated Adenylate Kinase 2 Deficiency Impairs Purine Metabolism and Ribosomal Biogenesis during Myelopoiesis

Wenqing Wang, Andrew Devilbiss, Martin Arreola, Thomas Mathews, Misty Martin-Sandoval, Zhiyu Zhao, Avni Awani, Daniel P. Dever, Matthew H. Porteus, Sean Morrison, Katja G Weinacht
Journal: Blood
Blood (2021) 138 (Supplement 1): 3257.
https://doi.org/10.1182/blood-2021-151919
Published: 2021
Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Suk See De Ravin, Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu, Cicera R. Lazzarotto, Taylor Liu, Sherry M. Koontz, Uimook Choi, Colin L. Sweeney, Narda Theobald, GaHyun Lee, Aaron B. Clark, Sandra S. Burkett, Benjamin P. Kleinstiver, Matthew H. Porteus, Shengdar Tsai, Douglas B. Kuhns, Gary A. Dahl, Stephen Headey, Xiaolin Wu, Harry L. Malech
Journal: Blood
Blood (2021) 137 (19): 2598–2608.
https://doi.org/10.1182/blood.2020008503
Published: 2021
Includes: Supplemental data
Journal Articles

Heterogeneity of Hematopoietic Stem and Progenitor Cell (HSPC) Composition in Αβ T-Cell/CD19 B-Cell Depleted Peripheral Blood Cell Stem Cell (PBSC) Transplant Grafts and Correlation with Immune and Hematopoietic Recovery

Giulia Barbarito, Beruch Dejene, BS, David C Shyr, MD, Gopin Saini, MBBS, Linda Oppizzi, BS, Sruthi Mantri, Hye-Sook Kwon, PhD, Matthew H. Porteus, MD PhD, Kenneth I. Weinberg, MD, Alice Bertaina, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 12–13.
https://doi.org/10.1182/blood-2020-143214
Published: 2020
Journal Articles

Reticular Dysgenesis-Associated Adenylate Kinase 2 Deficiency Impairs Hematopoietic Stem and Progenitor Cell Function through Reductive Stress

Wenqing Wang, PhD, Andrew Devilbiss, PhD, Thomas Mathews, PhD, Martin Arreola, Misty Martin, Zhiyu Zhao, DPhil, Avni Awani, PhD, Daniel P. Dever, PhD, Matthew H. Porteus, MD PhD, Sean Morrison, PhD, Katja G Weinacht, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 33.
https://doi.org/10.1182/blood-2020-138560
Published: 2020
Journal Articles

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Elie Haddad, Brent R. Logan, Linda M. Griffith, Rebecca H. Buckley, Roberta E. Parrott, Susan E. Prockop, Trudy N. Small, Jessica Chaisson, Christopher C. Dvorak, Megan Murnane, Neena Kapoor, Hisham Abdel-Azim, Imelda C. Hanson, Caridad Martinez, Jack J. H. Bleesing, Sharat Chandra, Angela R. Smith, Matthew E. Cavanaugh, Soma Jyonouchi, Kathleen E. Sullivan, Lauri Burroughs, Suzanne Skoda-Smith, Ann E. Haight, Audrey G. Tumlin, Troy C. Quigg, Candace Taylor, Blachy J. Dávila Saldaña, Michael D. Keller, Christine M. Seroogy, Kenneth B. Desantes, Aleksandra Petrovic, Jennifer W. Leiding, David C. Shyr, Hélène Decaluwe, Pierre Teira, Alfred P. Gillio, Alan P. Knutsen, Theodore B. Moore, Morris Kletzel, John A. Craddock, Victor Aquino, Jeffrey H. Davis, Lolie C. Yu, Geoffrey D. E. Cuvelier, Jeffrey J. Bednarski, Frederick D. Goldman, Elizabeth M. Kang, Evan Shereck, Matthew H. Porteus, James A. Connelly, Thomas A. Fleisher, Harry L. Malech, William T. Shearer, Paul Szabolcs, Monica S. Thakar, Mark T. Vander Lugt, Jennifer Heimall, Ziyan Yin, Michael A. Pulsipher, Sung-Yun Pai, Donald B. Kohn, Jennifer M. Puck, Morton J. Cowan, Richard J. O'Reilly, Luigi D. Notarangelo
Journal: Blood
Blood (2018) 132 (17): 1737–1749.
https://doi.org/10.1182/blood-2018-03-840702
Published: 2018
Includes: Supplemental data
Journal Articles

Knock-in editing: it functionally corrects!

Matthew H. Porteus
Journal: Blood
Blood (2016) 127 (21): 2507–2509.
https://doi.org/10.1182/blood-2016-03-703181
Published: 2016
Journal Articles

Ethical and regulatory aspects of genome editing

Clinical Trials & Observations
Donald B. Kohn, Matthew H. Porteus, Andrew M. Scharenberg
Journal: Blood
Blood (2016) 127 (21): 2553–2560.
https://doi.org/10.1182/blood-2016-01-678136
Published: 2016
Journal Articles

Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies

Ciaran Lee, PhD, Gang Bao, PhD, Matthew H. Porteus, MD PhD, Tatjana Cornu, PhD, Annarita Miccio, PhD, Thomas Cradick, Toni Cathomen, PhD, Ante Lundberg, MD, Fulvio Mavilio, PhD
Journal: Blood
Blood (2015) 126 (23): 3376.
https://doi.org/10.1182/blood.V126.23.3376.3376
Published: 2015
Journal Articles

Genome Engineering to Prospectively Investigate the Pathogenesis of MLL-AF9 Acute Leukemia

Corina Buechele, MD, Erin H. Breese, MDPhD, Dominik Schneidawind, MD, Matthew H. Porteus, MD PhD, Michael L. Cleary, MD
Journal: Blood
Blood (2014) 124 (21): 883.
https://doi.org/10.1182/blood.V124.21.883.883
Published: 2014
Journal Articles

Novel Integrated Autologous Hematopoietic Stem Cell Tracking in Nonhuman Primates Reveals Successive Pattern of Multi-Lineage Reconstitution after Total Body Irradiation

Jennifer E Adair, PhD, Shaina Porter, PhD, Kevin Haworth, PhD, Kenric Tam, BA, Hans-Peter Kiem, MD, Matthew H. Porteus, MD PhD
Journal: Blood
Blood (2014) 124 (21): 2910.
https://doi.org/10.1182/blood.V124.21.2910.2910
Published: 2014
Journal Articles

Using Genome Engineering To Prospectively Investigate The Pathogenesis Of MLL Translocations In Infant Acute Lymphoblastic Leukemia

Erin H. Breese, MD, PhD, Catherine Dawson, Corina Buechele, MD, Marcus R. Breese, PhD, Michael L. Cleary, MD, Matthew H. Porteus, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 3722.
https://doi.org/10.1182/blood.V122.21.3722.3722
Published: 2013
Journal Articles

Fine-Mapping and Genome Editing Reveal An Essential Erythroid Enhancer At The HbF-Associated BCL11A Locus

Daniel E. Bauer, MD, PHD, Sophia C. Kamran, MD, Samuel Lessard, BS, Jian Xu, PhD, Yuko Fujiwara, PhD, Carrie Lin, BS, Zhen Shao, PhD, Matthew C. Canver, BS, Elenoe C. Smith, PhD, Luca Pinello, PhD, Peter J. Sabo, PhD, Jeff Vierstra, BS, Richard A. Voit, BS, Guo-Cheng Yuan, PhD, Matthew H. Porteus, MD, PhD, John Stamatoyannopoulos, MD, Guillaume Lettre, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2013) 122 (21): 437.
https://doi.org/10.1182/blood.V122.21.437.437
Published: 2013
Journal Articles

Population Dynamics of Acute Myeloid Leukemia Clones by Barcode Tracking and High Throughput Sequencing

Christopher H. Hsu, MD, PhD, Shaina Porter, BS, Gareth Highnam, BS, David Mittelman, PhD, Matthew H. Porteus, MD, PhD
Journal: Blood
Blood (2012) 120 (21): 1398.
https://doi.org/10.1182/blood.V120.21.1398.1398
Published: 2012
Journal Articles

HbF-Associated Genetic Variation Marks an Erythroid Regulatory Element Essential for BCL11A Transcription and Subsequent Stage-Specific Globin Expression

Daniel E. Bauer, MD, PhD, Sophia C Kamran, B.S., Jian Xu, PhD, Carrie Lin, B.S., Zhen Shao, PhD, Yuko Fujiwara, PhD, Richard A. Voit, B.S., Matthew H. Porteus, MD, PhD, John A. Stamatoyannopoulos, MD, Stuart H. Orkin, MD
Journal: Blood
Blood (2012) 120 (21): 828.
https://doi.org/10.1182/blood.V120.21.828.828
Published: 2012