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Journal Articles

T-allo10 Immunotherapy Results in Enhanced Early Immune Reconstitution and Reduced GvHD with Excellent Clinical Outcomes Post Αβ-Depleted HSCT in Pediatric and Young Adult Hematologic Malignancy Patients

Alice Bertaina, Rosa Bacchetta, Alma-Martina Cepika, David C Shyr, Giulia Barbarito, Linda Oppizzi, Pauline Chen, Gopin Saini, Jennifer Lee, Karen Kristovich, Rajni Agarwal, Orly Klein, Kathryn Melsop, Keri Tate, Matthew Porteus, Maria Grazia Roncarolo

Blood (2024) 144 (Supplement 1): 3525.

https://doi.org/10.1182/blood-2024-194276

Published: 2024

Journal Articles

Evaluation of Bone Marrow in Fanconi Anemia Patients Treated with Briquilimab Antibody-Based Conditioning and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts

Charmaine Fay Soco, Mark R. Krampf, Yan Yi Chan, Hana Hoang, Nivedita Kunte, Gopin Saini, Kenneth I. Weinberg, Robertson Parkman, Alice Bertaina, Rajni Agarwal, Matthew Porteus, Agnieszka D. Czechowicz

Blood (2024) 144 (Supplement 1): 2015.

https://doi.org/10.1182/blood-2024-206381

Published: 2024

Journal Articles

Using Human Genetics to Develop Strategies to Increase Erythropoietic Output from Genome-Edited Hematopoietic Stem and Progenitor Cells

Sofia E Luna, Joab Camarena, Jessica P Hampton, Kiran R Majeti, Carsten T Charlesworth, Eric Soupene, Vivien A. Sheehan, Kyle Cromer, Matthew Porteus

Blood (2023) 142 (Supplement 1): 5003.

https://doi.org/10.1182/blood-2023-174170

Published: 2023

Journal Articles

Dual α-Globin and Truncated EPO Receptor Knockin Restores Hemoglobin Production in α-Thalassemia-Derived Hematopoietic Stem and Progenitor Cells

Simon N Chu, Eric Soupene, Beeke Wienert, Han Yin, Devesh Sharma, Kun Jia, Shota Homma, Jessica P Hampton, Bruce Conklin, Tippi C. MacKenzie, Matthew Porteus, Kyle Cromer

Blood (2023) 142 (Supplement 1): 485.

https://doi.org/10.1182/blood-2023-180187

Published: 2023

Journal Articles

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 5000.

https://doi.org/10.1182/blood-2023-188963

Published: 2023

Journal Articles

Radiation and Busulfan-Free Hematopoietic Stem Cell Transplantation Using Briquilimab (JSP191) Anti-CD117 Antibody-Conditioning, Transient Immunosuppression and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts in Patients with Fanconi Anemia

Blood (2023) 142 (Supplement 1): 229.

https://doi.org/10.1182/blood-2023-178400

Published: 2023

Journal Articles

Towards Automated Engineering of Donor-Derived T Lymphocytes into CRISPR/Cas9-Mediated CAR T Cells in a Closed-System

Aluya Richard Oseghale, Alice Bertaina, Matthew Porteus

Blood (2023) 142 (Supplement 1): 4826.

https://doi.org/10.1182/blood-2023-189883

Published: 2023

Journal Articles

Using Inducible Signaling Receptors to Increase Erythropoietic Output from Genome-Edited Hematopoietic Stem Cells

Kiran R Majeti, Aadit P Shah, Sofia E Luna, Eric Soupene, Simon N Chu, Devesh Sharma, Matthew Porteus, Kyle Cromer

Blood (2023) 142 (Supplement 1): 3627.

https://doi.org/10.1182/blood-2023-189791

Published: 2023

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

Blood (2019) 134 (Supplement_1): 3705.

https://doi.org/10.1182/blood-2019-129424

Published: 2019

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

Blood (2018) 132 (Supplement 1): 754.

https://doi.org/10.1182/blood-2018-99-119954

Published: 2018

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

Blood (2018) 132 (Supplement 1): 2558.

https://doi.org/10.1182/blood-2018-99-117926

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

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

Blood (2018) 132 (Supplement 1): 2192.

https://doi.org/10.1182/blood-2018-99-117371

Published: 2018

Journal Articles

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio

Blood (2018) 131 (17): 1960–1973.

https://doi.org/10.1182/blood-2017-10-811505

Published: 2018

Includes: Supplemental data

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

Blood (2017) 130 (Supplement 1): 3342.

https://doi.org/10.1182/blood.V130.Suppl_1.3342.3342

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

Blood (2017) 130 (Supplement 1): 4620.

https://doi.org/10.1182/blood.V130.Suppl_1.4620.4620

Published: 2017

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.

Blood (2017) 130 (Supplement 1): 284.

https://doi.org/10.1182/blood.V130.Suppl_1.284.284

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

Blood (2016) 128 (22): 1475.

https://doi.org/10.1182/blood.V128.22.1475.1475

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

Blood (2016) 128 (22): 5762.

https://doi.org/10.1182/blood.V128.22.5762.5762

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

Blood (2016) 128 (22): 321.

https://doi.org/10.1182/blood.V128.22.321.321

Published: 2016

NARROW

T-allo10 Immunotherapy Results in Enhanced Early Immune Reconstitution and Reduced GvHD with Excellent Clinical Outcomes Post Αβ-Depleted HSCT in Pediatric and Young Adult Hematologic Malignancy Patients

Evaluation of Bone Marrow in Fanconi Anemia Patients Treated with Briquilimab Antibody-Based Conditioning and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts

Using Human Genetics to Develop Strategies to Increase Erythropoietic Output from Genome-Edited Hematopoietic Stem and Progenitor Cells

Dual α-Globin and Truncated EPO Receptor Knockin Restores Hemoglobin Production in α-Thalassemia-Derived Hematopoietic Stem and Progenitor Cells

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Radiation and Busulfan-Free Hematopoietic Stem Cell Transplantation Using Briquilimab (JSP191) Anti-CD117 Antibody-Conditioning, Transient Immunosuppression and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts in Patients with Fanconi Anemia

Towards Automated Engineering of Donor-Derived T Lymphocytes into CRISPR/Cas9-Mediated CAR T Cells in a Closed-System

Using Inducible Signaling Receptors to Increase Erythropoietic Output from Genome-Edited Hematopoietic Stem Cells

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

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

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

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

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

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

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

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

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.

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

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

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

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American Society of Hematology

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American Society of Hematology

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