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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

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

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

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

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

Cytoskeletal abnormalities and neutrophil dysfunction in WDR1 deficiency

Blood (2016) 128 (17): 2135–2143.

https://doi.org/10.1182/blood-2016-03-706028

Published: 2016

Includes: Multimedia, Supplemental data

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

A Platform Minigene AAVS1 Targeted Safe Harbor Approach For Genetic Correction Of iPSC Derived From Patients With Each Of The 5 Genetic Forms Of Chronic Granulomatous Disease

Randall K Merling, Ph.D., Colin L Sweeney, Ph.D., Uimook Choi, Ph.D., Aaron Bodansky, Jessica Chu, Ph.D., Debra Long Priel, Douglas B Kuhns, Ph.D., Hongmei Wang, MD, Sam Vasilevsky, Ph.D., Thomas Winkler, MD, Suk See DeRavin, MD, Jizhong Zou, Ph.D., Thomas Leto, Ph.D., Cynthia E. Dunbar, MD, John Gallin, MD, Steven M. Holland, MD, Harry Malech, MD

Blood (2013) 122 (21): 1024.

https://doi.org/10.1182/blood.V122.21.1024.1024

Published: 2013

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

Journal Articles

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting

Jizhong Zou, Colin L. Sweeney, Bin-Kuan Chou, Uimook Choi, Jason Pan, Hongmei Wang, Sarah N. Dowey, Linzhao Cheng, Harry L. Malech

Blood (2011) 117 (21): 5561–5572.

https://doi.org/10.1182/blood-2010-12-328161

Published: 2011

Journal Articles

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

Elizabeth M. Kang, Uimook Choi, Narda Theobald, Gilda Linton, Debra A. Long Priel, Doug Kuhns, Harry L. Malech

Blood (2010) 115 (4): 783–791.

https://doi.org/10.1182/blood-2009-05-222760

Published: 2010

Includes: Supplemental data

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

Journal Articles

Methylguanine Methyltransferase-Based In Vivo Selection Results in Only Transient Improvement in Long-Term Marking after Autologous Transplantation of Transduced Hematopoietic Stem Cells in Rhesus Macaques.

Andre Larochelle, MD, PhD, Uimook Choi, Ph.D., Nora Naumann, Ph.D, Josh R. Clevenger, Harry L. Malech, M.D., Cynthia E. Dunbar, M.D.

Blood (2006) 108 (11): 3272.

https://doi.org/10.1182/blood.V108.11.3272.3272

Published: 2006

Journal Articles

WHIM syndrome myelokathexis reproduced in the NOD/SCID mouse xenotransplant model engrafted with healthy human stem cells transduced with C-terminus–truncated CXCR4

Toshinao Kawai, Uimook Choi, Lanise Cardwell, Suk See DeRavin, Nora Naumann, Narda L. Whiting-Theobald, Gilda F. Linton, Jaehyun Moon, Philip M. Murphy, Harry L. Malech

Blood (2006) 109 (1): 78–84.

https://doi.org/10.1182/blood-2006-05-025296

Published: 2006

Journal Articles

Correction of canine X-linked severe combined immunodeficiency by in vivo retroviral gene therapy

Suk See Ting–De Ravin, Douglas R. Kennedy, Nora Naumann, Jeffrey S. Kennedy, Uimook Choi, Brian J. Hartnett, Gilda F. Linton, Narda L. Whiting-Theobald, Peter F. Moore, William Vernau, Harry L. Malech, Peter J. Felsburg

Blood (2006) 107 (8): 3091–3097.

https://doi.org/10.1182/blood-2005-10-4057

Published: 2006

Journal Articles

Severe chronic graft-versus-host disease is characterized by a preponderance of CD4 + effector memory cells relative to central memory cells

Kouhei Yamashita, Uimook Choi, Patricia C. Woltz, Susan F. Foster, Michael C. Sneller, Francis T. Hakim, Daniel H. Fowler, Michael R. Bishop, Steven Z. Pavletic, Marisa Tamari, Kathleen Castro, A. John Barrett, Richard W. Childs, Gabor G. Illei, Susan F. Leitman, Harry L. Malech, Mitchell E. Horwitz

Blood (2004) 103 (10): 3986–3988.

https://doi.org/10.1182/blood-2003-09-3286

Published: 2004

NARROW

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

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

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

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

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

Cytoskeletal abnormalities and neutrophil dysfunction in WDR1 deficiency

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

A Platform Minigene AAVS1 Targeted Safe Harbor Approach For Genetic Correction Of iPSC Derived From Patients With Each Of The 5 Genetic Forms Of Chronic Granulomatous Disease

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

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

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).

Methylguanine Methyltransferase-Based In Vivo Selection Results in Only Transient Improvement in Long-Term Marking after Autologous Transplantation of Transduced Hematopoietic Stem Cells in Rhesus Macaques.

WHIM syndrome myelokathexis reproduced in the NOD/SCID mouse xenotransplant model engrafted with healthy human stem cells transduced with C-terminus–truncated CXCR4

Correction of canine X-linked severe combined immunodeficiency by in vivo retroviral gene therapy

Severe chronic graft-versus-host disease is characterized by a preponderance of CD4 + effector memory cells relative to central memory cells

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American Society of Hematology

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ASH Publications

American Society of Hematology

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