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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

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

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

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, PhD, Narda Theobald, Throm E Robert, PhD, John Gray, PhD, David J. Rawlings, MD, Brian P. Sorrentino, MD, Harry Malech, Suk See De Ravin, MD

Blood (2015) 126 (23): 2036.

https://doi.org/10.1182/blood.V126.23.2036.2036

Published: 2015

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

Improvement of Canine XSCID by In Vivo Gene Therapy Using RD114/TR-Pseudotyped SIV Lentiviral Vectors.

Morvarid Moayeri, MD, PhD, Suk See De Ravin, MD, PhD, Douglas R. Kennedy, BS, Nora Naumann, MD,PhD, Yasuhiro Ikeda, DVM,PhD, Peter J. Felsburg, VMD,PhD, Harry L. Malech, MD

Blood (2006) 108 (11): 3275.

https://doi.org/10.1182/blood.V108.11.3275.3275

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

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

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

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

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.

Improvement of Canine XSCID by In Vivo Gene Therapy Using RD114/TR-Pseudotyped SIV Lentiviral Vectors.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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