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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

Prime Editing Efficiently and Precisely Corrects Causative Mutation in Chronic Granulomatous Disease, Restoring Myeloid Function: Toward Development of a Prime Edited Autologous Hematopoietic Stem Cell Therapy

Jack M Heath, Jacob Stuart Orenstein, Justin G Tedeschi, Allen Ng, Maria D Collier, Julia Kushakji, Alan J Wilhelm, Andrew Taylor, David P Waterman, Suk See De Ravin, Harry L Malech, Andrew V Anzalone, Jeremy S Duffield, Jennifer L Gori

Blood (2023) 142 (Supplement 1): 7129.

https://doi.org/10.1182/blood-2023-186365

Published: 2023

Journal Articles

CRISPR/Cas9-mediated Cxcr4 disease allele inactivation for gene therapy in a mouse model of WHIM syndrome

Ji-Liang Gao, Albert Owusu-Ansah, Alexander Yang, Erin Yim, David H. McDermott, Paejonette Jacobs, Shamik Majumdar, Uimook Choi, Colin L. Sweeney, Harry L. Malech, Philip M. Murphy

Blood (2023) 142 (1): 23–32.

https://doi.org/10.1182/blood.2022019142

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

Extended Detection in Peripheral Blood Following Infusion of Chronic Granulomatous Disease Patient Autologous Granulocytes Corrected By mRNA Transfection in Patients with Chronic Granulomatous Disease

Vera Bzhilyanskaya, Ronald J. Meis, Narda Theobald, Tyra Estwick, Hong Lei, Steven Highfill, David F. Stroncek, Leonard Chen, Kamille West-Mitchell, Gary A. Dahl, Harry L Malech, Suk See De Ravin, Julie Brault

Blood (2022) 140 (Supplement 1): 5475–5476.

https://doi.org/10.1182/blood-2022-170390

Published: 2022

Journal Articles

Highly Efficient & Specific Repair of MAGT1 Mutation in Xmen Patient T Cells and Hematopoietic Stem Cells

Vera Bzhilyanskaya, Julie Brault, Siyuan Liu, Andres F. Zea Vera, Nikita Kozhushko, Amanda Lawson, Uimook Choi, Aaron B. Clark, Ronald J. Meis, Michelle Ma, Cicera Lazzarotto, Shengdar Tsai, Xiaolin Wu, Gary A. Dahl, Jenna Bergerson, Alexandra F. Freeman, Benjamin Kleinstiver, Harry L Malech, Suk See De Ravin

Blood (2022) 140 (Supplement 1): 7766–7767.

https://doi.org/10.1182/blood-2022-170252

Published: 2022

Journal Articles

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Blood (2021) 138 (26): 2768–2780.

https://doi.org/10.1182/blood.2021011192

Published: 2021

Includes: Supplemental data

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

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

Blood (2021) 137 (19): 2598–2608.

https://doi.org/10.1182/blood.2020008503

Published: 2021

Includes: Supplemental data

Journal Articles

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Blood (2019) 134 (Supplement_1): 2058.

https://doi.org/10.1182/blood-2019-126746

Published: 2019

Journal Articles

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

Julie Brault, PhD PharmD, Ezekiel Bello, Ronald J. Meis, PhD, Taylor Liu, Sherry M. Koontz, BS, Kennichi Dowdell, PhD, Linhong Li, PhD, Cornell Allen, MS, Narda Theobald, BS, Janet Lee, BS, Aaron B. Clark, BS, Juan C. Ravell, MD, Michael Lenardo, MD, Gary A. Dahl, PhD, Harry L. Malech, MD, Suk See De Ravin, MD, PhD

Blood (2019) 134 (Supplement_1): 4637.

https://doi.org/10.1182/blood-2019-129062

Published: 2019

Journal Articles

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Naoya Uchida, MD, Linhong Li, PhD, Tina Nassehi, Morgan Yapundich, Jackson Gamer, Claire Drysdale, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Cornell Allen, MS, Suk See De Ravin, MD, PhD, Robert E. Donahue, VMD, Harry L. Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 609.

https://doi.org/10.1182/blood-2019-122727

Published: 2019

Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

Published: 2019

Journal Articles

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

Naoya Uchida, MD, Linhong Li, Juan J Haro-Mora, PhD, Selami Demirci, PhD, Tina Nassehi, Jackson Gamer, Claire Drysdale, Morgan Yapundich, Cornell Allen, Suk See De Ravin, MD PhD, Harry L Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2018) 132 (Supplement 1): 2191.

https://doi.org/10.1182/blood-2018-99-111677

Published: 2018

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

Blood (2018) 132 (17): 1737–1749.

https://doi.org/10.1182/blood-2018-03-840702

Published: 2018

Includes: Supplemental data

Journal Articles

Aberrant Clonal Hematopoiesis of the Erythroid and Myeloid Lineages in a Lentivirally Barcoded Rhesus Macaque

Diego A Espinoza, BA, Chuanfeng Wu, MD, Xing Fan, MD, Di Yang, MD, Idalia Montano Yabe, MS, Selami Demirci, PhD, So Gun Hong, PhD, Aylin Bonifacino, MS, Allen Krouse, Mark Metzger, Rong Lu, PhD, Katherine R Calvo, MD PhD, John F Tisdale, MD, Xiaolin Wu, PhD, Suk See De Ravin, MD PhD, Harry L Malech, MD, Robert E Donahue, VMD, Cynthia E Dunbar, MD

Blood (2017) 130 (Supplement 1): 606.

https://doi.org/10.1182/blood.V130.Suppl_1.606.606

Published: 2017

Journal Articles

Enhanced Expression of CXCR4 Facilitates the Trafficking of Anti-c-Kit Chimeric Antigen Receptor (CAR) T Cells to Bone Marrow and Achieves Effective Donor Stem Cell Engraftment

Yasuyuki Arai, MDPhD, Cristina I. Corsino, Sherry M. Koontz, Masaki Tajima, Mary A. Black, Colin L. Sweeney, Uimook Choi, Steven A. Feldman, Mary C. Dinauer, Harry L Malech, MD

Blood (2017) 130 (Supplement 1): 4446.

https://doi.org/10.1182/blood.V130.Suppl_1.4446.4446

Published: 2017

Journal Articles

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Blood (2017) 130 (Supplement 1): 523.

https://doi.org/10.1182/blood.V130.Suppl_1.523.523

Published: 2017

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

Prime Editing Efficiently and Precisely Corrects Causative Mutation in Chronic Granulomatous Disease, Restoring Myeloid Function: Toward Development of a Prime Edited Autologous Hematopoietic Stem Cell Therapy

CRISPR/Cas9-mediated Cxcr4 disease allele inactivation for gene therapy in a mouse model of WHIM syndrome

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

Extended Detection in Peripheral Blood Following Infusion of Chronic Granulomatous Disease Patient Autologous Granulocytes Corrected By mRNA Transfection in Patients with Chronic Granulomatous Disease

Highly Efficient & Specific Repair of MAGT1 Mutation in Xmen Patient T Cells and Hematopoietic Stem Cells

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

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

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

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

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

Aberrant Clonal Hematopoiesis of the Erythroid and Myeloid Lineages in a Lentivirally Barcoded Rhesus Macaque

Enhanced Expression of CXCR4 Facilitates the Trafficking of Anti-c-Kit Chimeric Antigen Receptor (CAR) T Cells to Bone Marrow and Achieves Effective Donor Stem Cell Engraftment

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

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American Society of Hematology

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ASH Publications

American Society of Hematology

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