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Journal Articles

Human Gene Therapy Optimized for the Platelet Bleeding Disorder Glanzmann Thrombasthenia

Juan Fang, Matthew Hogenauer, Aoife M. Roche, Sevasti B. Koukouritaki, Lily M. Du, Huiqing Xu, John Everett, Frederic D. Bushman, Myriam Armant, Peiman Hematti, David A. Wilcox

Blood (2024) 144 (Supplement 1): 5409.

https://doi.org/10.1182/blood-2024-199243

Published: 2024

Journal Articles

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Blood (2024) 144 (15): 1633–1645.

https://doi.org/10.1182/blood.2023023349

Published: 2024

Includes: Supplemental data

Journal Articles

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

David A. Wilcox, Myriam Armant, Lynn Malec, Qizhen Shi, Bryon D. Johnson, Huiqing Xu, Lily M. Du, Lisa J. Duffy, Frederic D. Bushman, James H. Jerkins, Peiman Hematti, Ruta Brazauskas, Shawn M. Jobe, Parameswaran N. Hari, Mary Eapen

Blood (2023) 142 (Supplement 1): 2249.

https://doi.org/10.1182/blood-2023-172845

Published: 2023

Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Blood (2023) 142 (15): 1281–1296.

https://doi.org/10.1182/blood.2022019117

Published: 2023

Includes: Supplemental data

Journal Articles

Chemotherapy-induced reversal of ciltacabtagene autoleucel–associated movement and neurocognitive toxicity

Charlotte E. Graham, Won-Ho Lee, Hadley R. Wiggin, Valentina M. Supper, Mark B. Leick, Filippo Birocchi, Andrew J. Yee, Angelina Petrichenko, John Everett, Frederic D. Bushman, Hossein Sadrzadeh, Otto Rapalino, Daniel Chiu, Isabel Arrillaga-Romany, Marcela V. Maus, Matthew J. Frigault, Kathleen M. E. Gallagher

Blood (2023) 142 (14): 1248–1252.

https://doi.org/10.1182/blood.2023021429

Published: 2023

Includes: Supplemental data

Journal Articles

Novel extragenic genomic safe harbors for precise therapeutic T-cell engineering

Ashlesha Odak, Han Yuan, Judith Feucht, Vito Adrian Cantu, Jorge Mansilla-Soto, Friederike Kogel, Justin Eyquem, John Everett, Frederic D. Bushman, Christina S. Leslie, Michel Sadelain

Blood (2023) 141 (22): 2698–2712.

https://doi.org/10.1182/blood.2022018924

Published: 2023

Includes: Supplemental data

Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Blood (2022) 140 (Supplement 1): 10643–10645.

https://doi.org/10.1182/blood-2022-159768

Published: 2022

Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Blood (2022) 140 (Supplement 1): 7770–7771.

https://doi.org/10.1182/blood-2022-159979

Published: 2022

Journal Articles

Decade-Long Remissions of Leukemia Sustained By the Persistence of Activated CD4+ CAR T-Cells

Blood (2021) 138 (Supplement 1): 166.

https://doi.org/10.1182/blood-2021-152664

Published: 2021

Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Blood (2021) 138 (Supplement 1): 2934.

https://doi.org/10.1182/blood-2021-147697

Published: 2021

Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Blood (2021) 138 (15): 1304–1316.

https://doi.org/10.1182/blood.2020010260

Published: 2021

Includes: Supplemental data

Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

Journal Articles

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Blood (2019) 134 (Supplement_1): 49.

https://doi.org/10.1182/blood-2019-122374

Published: 2019

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

Long-Term Remission of CLL Sustained By Pauciclonal Anti-CD19 Chimeric Antigen Receptor T (CTL019) Cell Clones

J. Joseph Melenhorst, PhD, David L. Porter, MD, Lifeng Tian, PhD, Simon F Lacey, PhD BS, Christopher L Nobles, PhD, Joseph A Fraietta, PhD, Noelle V. Frey, MD, Irina Kulikovskaya, Minnal Gupta, Regina M Young, PhD, David E Ambrose, BS, Don L. Siegel, MD PhD, Frederic D Bushman, PhD, Carl H June, MD

Blood (2018) 132 (Supplement 1): 699.

https://doi.org/10.1182/blood-2018-99-117390

Published: 2018

Journal Articles

Vector Integration and Efficacy of CD19-Directed CAR T Cell Therapy in Acute Lymphoblastic Leukemia (ALL) and Chronic Lymphocytic Leukemia (CLL)

Christopher L Nobles, PhD, John K Everett, Shantan Reddy, Joseph A Fraietta, PhD, David L. Porter, MD, Noelle V. Frey, MD, Stephan A. Grupp, MD PhD, Don L. Siegel, MD PhD, Simon F Lacey, PhD BS, Carl H June, MD, Frederic D Bushman, PhD, J. Joseph Melenhorst, PhD

Blood (2018) 132 (Supplement 1): 4548.

https://doi.org/10.1182/blood-2018-99-117034

Published: 2018

Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Blood (2018) 132 (Supplement 1): 1021.

https://doi.org/10.1182/blood-2018-99-119591

Published: 2018

Journal Articles

Clinical Evidence That Deficiency of the Methylcytosine Dioxygenase TET2 Gene Promotes the Clonal Expansion and Therapeutic Efficacy of CD19-Directed T Cells

Blood (2017) 130 (Supplement 1): 1909.

https://doi.org/10.1182/blood.V130.Suppl_1.1909.1909

Published: 2017

Journal Articles

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Blood (2017) 130 (Supplement 1): 4618.

https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618

Published: 2017

NARROW

Human Gene Therapy Optimized for the Platelet Bleeding Disorder Glanzmann Thrombasthenia

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Chemotherapy-induced reversal of ciltacabtagene autoleucel–associated movement and neurocognitive toxicity

Novel extragenic genomic safe harbors for precise therapeutic T-cell engineering

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Decade-Long Remissions of Leukemia Sustained By the Persistence of Activated CD4+ CAR T-Cells

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Long-Term Remission of CLL Sustained By Pauciclonal Anti-CD19 Chimeric Antigen Receptor T (CTL019) Cell Clones

Vector Integration and Efficacy of CD19-Directed CAR T Cell Therapy in Acute Lymphoblastic Leukemia (ALL) and Chronic Lymphocytic Leukemia (CLL)

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Clinical Evidence That Deficiency of the Methylcytosine Dioxygenase TET2 Gene Promotes the Clonal Expansion and Therapeutic Efficacy of CD19-Directed T Cells

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

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American Society of Hematology

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ASH Publications

American Society of Hematology

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