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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

Clinical Characteristics, Management, and Allogeneic Hematopoietic Cell Transplantation of Patients with Toll-like Receptor 8 Gain-of-Function Mutations

Danielle E Arnold, Saara Kaviany, Jahnavi Aluri, Luis Murguia-Favela, Nicola A. Wright, Thomas P. Loughran, Jr., Jonathan Powell, Suk See De Ravin, V. Koneti Rao, Steven Holland, Jack Bleesing, Jeffrey J. Bednarski, James A. Connelly, Megan Cooper

Blood (2023) 142 (Supplement 1): 936.

https://doi.org/10.1182/blood-2023-185110

Published: 2023

Journal Articles

The promise of in vivo HSC prime editing

Colin L. Sweeney, Suk See De Ravin

Blood (2023) 141 (17): 2039–2040.

https://doi.org/10.1182/blood.2023019922

Published: 2023

Journal Articles

Clonal Dynamics of HDR-Edited HSPCs Targeting the CD33 Locus in Rhesus Macaques

Byung-Chul Lee, Max Grice, Chuanfeng Wu, Ryland D Mortlock, Taehoon Shin, Yifan Zhou, Uimook Choi, Suk See De Ravin, So Gun Hong, Cynthia E. Dunbar

Blood (2022) 140 (Supplement 1): 309–310.

https://doi.org/10.1182/blood-2022-166504

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

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

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

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

Blood (2021) 137 (18): 2450–2462.

https://doi.org/10.1182/blood.2020009620

Published: 2021

Includes: Supplemental data

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

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

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

Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34 + cells

Randall K. Merling, Colin L. Sweeney, Uimook Choi, Suk See De Ravin, Timothy G. Myers, Francisco Otaizo-Carrasquero, Jason Pan, Gilda Linton, Lifeng Chen, Sherry Koontz, Narda L. Theobald, Harry L. Malech

Blood (2013) 121 (14): e98–e107.

https://doi.org/10.1182/blood-2012-03-420273

Published: 2013

NARROW

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

Clinical Characteristics, Management, and Allogeneic Hematopoietic Cell Transplantation of Patients with Toll-like Receptor 8 Gain-of-Function Mutations

The promise of in vivo HSC prime editing

Clonal Dynamics of HDR-Edited HSPCs Targeting the CD33 Locus in Rhesus Macaques

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

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

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

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

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

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

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

Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34 + cells

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American Society of Hematology

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ASH Publications

American Society of Hematology

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