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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 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

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

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

Journal Articles

The Contribution of the Thymus to Autoimmune Phenotypes in GvHD and Colitis

Karlie R. Sharma, PhD, Sarah E Bryant, BS, Sherry Koontz, Harry L. Malech, MD, Elizabeth M. Kang, MD

Blood (2016) 128 (22): 3347.

https://doi.org/10.1182/blood.V128.22.3347.3347

Published: 2016

Journal Articles

Whim Syndrome Caused By a 5 Base Pair Deletion in the C-Terminus of Chemokine Receptor CXCR4

Qian Liu, MD, Lizbeeth Lopez, Catherina Pan, Daniel Velez, John S. Corns, Donald T. Ellenburg, Harry L. Malech, MD, Philip M. Murphy, MD, David H. McDermott, MD

Blood (2015) 126 (23): 1003.

https://doi.org/10.1182/blood.V126.23.1003.1003

Published: 2015

Journal Articles

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MDPhD, Xiaolin Wu, Narda Theobald, Janet S Lee, John Gray, PhD, Imelda Celine Hanson, MD, Luigi Notarangelo, MDPhD, Brian P. Sorrentino, MD, Harry L. Malech, MD

Blood (2015) 126 (23): 261.

https://doi.org/10.1182/blood.V126.23.261.261

Published: 2015

Journal Articles

Graft Versus Host Disease (GvHD) Is Attenuated By Administration of Pregnancy Specific Glycoproteins through Induction of Immune Tolerance

Karlie R. Sharma, PhD, Shemona Rattila, Patricia Kiesler, PhD, Angela Ballesteros, PhD, Jian Luo, PhD, Sherry Koontz, James Warren, MS, Harry L. Malech, MD, Gabriela Dveksler, PhD, Elizabeth M. Kang, MD

Blood (2015) 126 (23): 4278.

https://doi.org/10.1182/blood.V126.23.4278.4278

Published: 2015

Journal Articles

Allogeneic Transplantation for P40 CGD Is Curative of Inflammatory Disease

Elizabeth M. Kang, MD, Nicola A. Wright, MD FRCPC, Katherine Nicholls, MBBS, FRACP, FRCPA, Dianne Hilligoss, RN, MSN, CRNP, Corin Kelly, RN, Douglas B. Kuhns, PhD, Harry L. Malech, MD

Blood (2015) 126 (23): 5515.

https://doi.org/10.1182/blood.V126.23.5515.5515

Published: 2015

Journal Articles

Adenosine A 2A Receptor Agonist Mediated Prevention of Graft-Versus Host Disease In the Mouse Is Associated with An Increase of T-Regulatory Cells In Target Organs.

Kyu Lee Han, PhD, Sherry Walker, Harry L. Malech, MD, Elizabeth M. Kang, MD

Blood (2010) 116 (21): 3744.

https://doi.org/10.1182/blood.V116.21.3744.3744

Published: 2010

Journal Articles

Successful Control of Preexistent Active Infection by Granulocyte Transfusions During Conditioning Induced Cytopenia In Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Stem Cell Transplant

P. Dayand Borge, Jr., MD, Narda Theobald, MA, Rosamma DeCastro, NP, Harry L. Malech, MD, Susan Leitman, MD, Elizabeth M. Kang, MD

Blood (2010) 116 (21): 1329.

https://doi.org/10.1182/blood.V116.21.1329.1329

Published: 2010

Journal Articles

Activation of the Adenosine A 2A Receptor Promotes the Development of Donor-Derived T Regulatory Cells in a Graft-Versus Host Disease Mouse Model.

Kyu Lee Han, PhD, Cattlena M. Changpriroa, Harry L. Malech, MD, Elizabeth M. Kang, MD

Blood (2009) 114 (22): 1339.

https://doi.org/10.1182/blood.V114.22.1339.1339

Published: 2009

Journal Articles

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

Elizabeth M. Kang, MD, Gilda Linton, Narda Theobald, Sandra Anaya O’Brien, RN, Dianne Hilligoss, CNP, Harry L. Malech, MD

Blood (2007) 110 (11): 504.

https://doi.org/10.1182/blood.V110.11.504.504

Published: 2007

Journal Articles

Correction of Human X-Linked Chronic Granulomatous Disease (X-CGD) Following Transduction of X-CGD CD34+ Cells with a Modified RD114-Pseudotyped Simian Immunodeficiency Virus Lentiviral Vector in a NOD/SCID Mouse Xenograft Model.

Nora Naumann, MD, PhD, Suk See De Ravin, MD, PhD, Uimook Choi, PhD, Morvarid Moayeri, MD, PhD, Yasuhiro Ikeda, DVM, PhD, Harry L. Malech, MD

Blood (2006) 108 (11): 3278.

https://doi.org/10.1182/blood.V108.11.3278.3278

Published: 2006

Journal Articles

Enhanced Engraftment of Human CD34+ Stem Cells in NOD/SCID Mice by Diprotin A Occurs by Inhibition of Recipient CD26/Dipeptidyl Peptidase-IV (DPP-IV).

Toshinao Kawai, MD, Uimook Choi, PhD, Po-Ching Liu, PhD, Harry L. Malech, MD

Blood (2006) 108 (11): 3178.

https://doi.org/10.1182/blood.V108.11.3178.3178

Published: 2006

NARROW

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 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

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

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

The Contribution of the Thymus to Autoimmune Phenotypes in GvHD and Colitis

Whim Syndrome Caused By a 5 Base Pair Deletion in the C-Terminus of Chemokine Receptor CXCR4

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Graft Versus Host Disease (GvHD) Is Attenuated By Administration of Pregnancy Specific Glycoproteins through Induction of Immune Tolerance

Allogeneic Transplantation for P40 CGD Is Curative of Inflammatory Disease

Adenosine A 2A Receptor Agonist Mediated Prevention of Graft-Versus Host Disease In the Mouse Is Associated with An Increase of T-Regulatory Cells In Target Organs.

Successful Control of Preexistent Active Infection by Granulocyte Transfusions During Conditioning Induced Cytopenia In Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Stem Cell Transplant

Activation of the Adenosine A 2A Receptor Promotes the Development of Donor-Derived T Regulatory Cells in a Graft-Versus Host Disease Mouse Model.

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

Correction of Human X-Linked Chronic Granulomatous Disease (X-CGD) Following Transduction of X-CGD CD34+ Cells with a Modified RD114-Pseudotyped Simian Immunodeficiency Virus Lentiviral Vector in a NOD/SCID Mouse Xenograft Model.

Enhanced Engraftment of Human CD34+ Stem Cells in NOD/SCID Mice by Diprotin A Occurs by Inhibition of Recipient CD26/Dipeptidyl Peptidase-IV (DPP-IV).

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American Society of Hematology

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ASH Publications

American Society of Hematology

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