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Journal Articles

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

Blood (2024) 144 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2024-202543

Published: 2024

Journal Articles

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Danielle E. Arnold, Suhag Parikh, Brent Logan, Rebecca A. Marsh, Linda M. Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J. Si Lim, Eyal Grunebaum, E. Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K. Prasad, Jennifer R. Heimall, Fabien Touzot, Lauri M. Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R. Oshrine, Jeffrey J. Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D. E. Cuvelier, Lisa R. Forbes Satter, Caridad Martinez, Mark T. Vander Lugt, Lolie C. Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E. Prockop, Blachy J. Dávila Saldaña, Victor Aquino, Larisa A. Broglie, Christen L. Ebens, Lisa M. Madden, Kenneth DeSantes, Jordan Milner, Hemalatha G. Rangarajan, Ami J. Shah, Alfred P. Gillio, Alan P. Knutsen, Holly K. Miller, Theodore B. Moore, Pamela Graham, Andrea Bauchat, Nancy J. Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J. Dorsey, Olga Birbrayer, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson, Harry L. Malech, Elizabeth M. Kang

Blood (2023) 142 (24): 2105–2118.

https://doi.org/10.1182/blood.2022019586

Published: 2023

Includes: Supplemental data

Journal Articles

Α-Globin Lentiviral Vectors for Hematopoietic Stem Cell Gene Therapy of α-Thalassemia

Eva Segura, Kevin Tam, Beatriz Campo Fernandez, Tippi C. MacKenzie, Roger Hollis, Donald B. Kohn

Blood (2023) 142 (Supplement 1): 7131.

https://doi.org/10.1182/blood-2023-190619

Published: 2023

Journal Articles

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Lauren Jimenez-Kurlander, Pei-Chi Kao, Mira Magner, Emily Morris, Amy Federico, Theodore Moore, Satiro DeOliveira, Augustine Fernandes, Donald B. Kohn, Wendy B. London, David A. Williams, Erica B. Esrick

Blood (2023) 142 (Supplement 1): 3750.

https://doi.org/10.1182/blood-2023-186915

Published: 2023

Journal Articles

Autologous MPO Knock-out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension

Aurélien BL Colamartino, Zulema Romero Garcia, Jenny Shapiro, Anat Kahana, Yeshai Schary, Leoni Gnatzy-Feik, Eva Berghausen, Mario Zierden, Stephan Rosenkranz, Donald B. Kohn

Blood (2023) 142 (Supplement 1): 7120.

https://doi.org/10.1182/blood-2023-178808

Published: 2023

Journal Articles

β-Thalassemia: all about that base, no cutting

Paul G. Ayoub, Donald B. Kohn

Blood (2023) 141 (10): 1098–1099.

https://doi.org/10.1182/blood.2022019350

Published: 2023

Journal Articles

Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression

Ryan L. Wong, Sarah Sackey, Devin Brown, Shantha Senadheera, Katelyn Masiuk, Jason P. Quintos, Nicole Colindres, Luke Riggan, Richard A. Morgan, Harry L. Malech, Roger P. Hollis, Donald B. Kohn

Blood (2023) 141 (9): 1007–1022.

https://doi.org/10.1182/blood.2022016074

Published: 2023

Includes: Supplemental data

Journal Articles

Base Editing of Hematopoietic Stem Cells Rescues T-Cell Development for CD3d Severe Combined Immunodeficiency

Grace McAuley, Gloria Yiu, Gregory A. Newby, Beatriz Campo Fernandez, Xiaomeng Wu, Patrick Chang, Kelcee Everette, Zulema Romero Garcia, David R. Liu, Gay M. Crooks, Donald B. Kohn

Blood (2022) 140 (Supplement 1): 1641–1642.

https://doi.org/10.1182/blood-2022-170634

Published: 2022

Journal Articles

Combining the Anti-Sickling βAS3-Globin Gene with BCL11A and ZNF410 Double Shmirs for the Treatment of Sickle Cell Disease

Boya Liu, Kevyn Hart, Christian Brendel, Donald B. Kohn, David A. Williams

Blood (2022) 140 (Supplement 1): 7786–7787.

https://doi.org/10.1182/blood-2022-163469

Published: 2022

Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Blood (2022) 140 (Supplement 1): 7774–7775.

https://doi.org/10.1182/blood-2022-158578

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

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Blood (2022) 140 (Supplement 1): 10665–10667.

https://doi.org/10.1182/blood-2022-169089

Published: 2022

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

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier, Brent R. Logan, Susan E. Prockop, Rebecca H. Buckley, Caroline Y. Kuo, Linda M. Griffith, Xuerong Liu, Alison Yip, Michael S. Hershfield, Paul G. Ayoub, Theodore B. Moore, Morna J. Dorsey, Richard J. O’Reilly, Neena Kapoor, Sung-Yun Pai, Malika Kapadia, Christen L. Ebens, Lisa R. Forbes Satter, Lauri M. Burroughs, Aleksandra Petrovic, Deepak Chellapandian, Jennifer Heimall, David C. Shyr, Ahmad Rayes, Jeffrey J. Bednarski, Sharat Chandra, Shanmuganathan Chandrakasan, Alfred P. Gillio, Lisa Madden, Troy C. Quigg, Emi H. Caywood, Blachy J. Dávila Saldaña, Kenneth DeSantes, Hesham Eissa, Frederick D. Goldman, Jacob Rozmus, Ami J. Shah, Mark T. Vander Lugt, Monica S. Thakar, Roberta E. Parrott, Caridad Martinez, Jennifer W. Leiding, Troy R. Torgerson, Michael A. Pulsipher, Luigi D. Notarangelo, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Jennifer M. Puck, Donald B. Kohn

Blood (2022) 140 (7): 685–705.

https://doi.org/10.1182/blood.2022016196

Published: 2022

Includes: Supplemental data

Journal Articles

AML Cell Vaccines Co-Expressing CD80 and IL-15/IL-15 Receptor Alpha Induce Activation and Cytolytic Activity in Post Remission Autologous Patient PBMC Ex Vivo

Xinyue Wang, Jeffrey P. Fung, Giulia Parisi, Francesca M. Olguin, Nathaniel Z. Rothschild, Kenneth I. Weinberg, William J. Murphy, Donald B. Kohn, Karin L. Gaensler

Blood (2021) 138 (Supplement 1): 1706.

https://doi.org/10.1182/blood-2021-151614

Published: 2021

Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz

Blood (2021) 138 (Supplement 1): 2932.

https://doi.org/10.1182/blood-2021-151898

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

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD, Gayatri R Rao, MD JD, Elena Almarza, PhD, Dayna Terrazas, RN, Eileen Nicoletti, MD, Augustine Fernandes, PhD, Caroline Kuo, MD, Satiro N. De Oliveira, MD, Theodore B. Moore, MD, Kenneth M Law, PhD, Brian C Beard, PhD, Julian Sevilla, MD PhD, Cristina Mesa-Núñez, PhD, Juan A Bueren, PhD, Jonathan D Schwartz, MD

Blood (2020) 136 (Supplement 1): 15.

https://doi.org/10.1182/blood-2020-142484

Published: 2020

Journal Articles

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

Georgia L. Gregory, BS, Beeke Wienert, PhD, Marisa Schwab, MD, Billie Rachael Lianoglou, MS, Roger P. Hollis, PhD, Donald B. Kohn, MD, Bruce R. Conklin, MD, Tippi C. MacKenzie, MD

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-142922

Published: 2020

Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-137762

Published: 2020

NARROW

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Α-Globin Lentiviral Vectors for Hematopoietic Stem Cell Gene Therapy of α-Thalassemia

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Autologous MPO Knock-out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension

β-Thalassemia: all about that base, no cutting

Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression

Base Editing of Hematopoietic Stem Cells Rescues T-Cell Development for CD3d Severe Combined Immunodeficiency

Combining the Anti-Sickling βAS3-Globin Gene with BCL11A and ZNF410 Double Shmirs for the Treatment of Sickle Cell Disease

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

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

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

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

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

AML Cell Vaccines Co-Expressing CD80 and IL-15/IL-15 Receptor Alpha Induce Activation and Cytolytic Activity in Post Remission Autologous Patient PBMC Ex Vivo

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

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

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

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American Society of Hematology

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American Society of Hematology

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