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Malech, Harry Lewis
Journal title:American Men & Women of Science : A Biographical Directory of Today’s Leaders in Physical, Biological and Related Sciences: LIT-MEK, 2022, p.7214-7214
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Malech, Harry Lewis
Journal title:American Men & Women of Science : A Biographical Directory of Today’s Leaders in Physical, Biological and Related Sciences: LIU-MEM, 2021, p.7188-7188
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ArticlesPeer ReviewedFull Text
Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle
Author / Creator:Malech, Harry L
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2021-01, Vol.384 (3), p.286-287
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Neutrophil extracellular traps enriched in oxidized mitochondrial DNA are interferogenic and contribute to lupus-like disease
Author / Creator:Lood, Christian ; Blanco, Luz P ; Purmalek, Monica M ; Carmona-Rivera, Carmelo ; De Ravin, Suk S ; Smith, Carolyne K ; Malech, Harry L ; Ledbetter, Jeffrey A ; Elkon, Keith B ; Kaplan, Mariana J
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2016-02, Vol.22 (2), p.146-153
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Differentiation of human and murine induced pluripotent stem cells to microglia-like cells
Author / Creator:Pandya, Hetal ; Shen, Michael J ; Ichikawa, David M ; Sedlock, Andrea B ; Choi, Yong ; Johnson, Kory R ; Kim, Gloria ; Brown, Mason A ; Elkahloun, Abdel G ; Maric, Dragan ; Sweeney, Colin L ; Gossa, Selamawit ; Malech, Harry L ; McGavern, Dorian B ; Park, John K
Publisher:New York: Nature Publishing Group US
Journal title:Nature neuroscience, 2017-05, Vol.20 (5), p.753-759
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Gene correction for SCID-X1 in long-term hematopoietic stem cells
Author / Creator:Pavel-Dinu, Mara ; Wiebking, Volker ; Dejene, Beruh T. ; Srifa, Waracharee ; Mantri, Sruthi ; Nicolas, Carmencita E. ; Lee, Ciaran ; Bao, Gang ; Kildebeck, Eric J. ; Punjya, Niraj ; Sindhu, Camille ; Inlay, Matthew A. ; Saxena, Nivedita ; DeRavin, Suk See ; Malech, Harry ; Roncarolo, Maria Grazia ; Weinberg, Kenneth I. ; Porteus, Matthew H.
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2019-04, Vol.10 (1), p.1634-1634, Article 1634
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Lentiviral Gene Therapy for Artemis-Deficient SCID
Author / Creator:Cowan, Morton J. ; Yu, Jason ; Facchino, Janelle ; Fraser-Browne, Carol ; Sanford, Ukina ; Kawahara, Misako ; Dara, Jasmeen ; Long-Boyle, Janel ; Oh, Jess ; Chan, Wendy ; Chag, Shivali ; Broderick, Lori ; Chellapandian, Deepak ; Decaluwe, Hélène ; Golski, Catherine ; Hu, Diana ; Kuo, Caroline Y. ; Miller, Holly K. ; Petrovic, Aleksandra ; Currier, Robert ; Hilton, Joan F. ; Punwani, Divya ; Dvorak, Christopher C. ; Malech, Harry L. ; McIvor, R. Scott ; Puck, Jennifer M.
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2022-12, Vol.387 (25), p.2344-2355
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Pyocyanin-enhanced neutrophil extracellular trap formation requires the NADPH oxidase
Author / Creator:Rada, Balázs ; Jendrysik, Meghan A ; Pang, Lan ; Hayes, Craig P ; Yoo, Dae-Goon ; Park, Jonathan J ; Moskowitz, Samuel M ; Malech, Harry L ; Leto, Thomas L
Publisher:United States: Public Library of Science
Journal title:PloS one, 2013-01, Vol.8 (1), p.e54205-e54205
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Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease
Author / Creator:De Ravin, Suk See ; Reik, Andreas ; Liu, Pei-Qi ; Li, Linhong ; Wu, Xiaolin ; Su, Ling ; Raley, Castle ; Theobald, Narda ; Choi, Uimook ; Song, Alexander H ; Chan, Andy ; Pearl, Jocelynn R ; Paschon, David E ; Lee, Janet ; Newcombe, Hannah ; Koontz, Sherry ; Sweeney, Colin ; Shivak, David A ; Zarember, Kol A ; Peshwa, Madhusudan V ; Gregory, Philip D ; Urnov, Fyodor D ; Malech, Harry L
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2016-04, Vol.34 (4), p.424-429
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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency
Author / Creator:De Ravin, Suk See ; Liu, Siyuan ; Sweeney, Colin L. ; Brault, Julie ; Whiting-Theobald, Narda ; Ma, Michelle ; Liu, Taylor ; Choi, Uimook ; Lee, Janet ; O’Brien, Sandra Anaya ; Quackenbush, Priscilla ; Estwick, Tyra ; Karra, Anita ; Docking, Ethan ; Kwatemaa, Nana ; Guo, Shuang ; Su, Ling ; Sun, Zhonghe ; Zhou, Sheng ; Puck, Jennifer ; Cowan, Morton J. ; Notarangelo, Luigi D. ; Kang, Elizabeth ; Malech, Harry L. ; Wu, Xiaolin
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2022-06, Vol.13 (1), p.3710-3710, Article 3710
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ArticlesPeer ReviewedFull Text
Residual NADPH Oxidase and Survival in Chronic Granulomatous Disease
Author / Creator:Kuhns, Douglas B ; Alvord, W. Gregory ; Heller, Theo ; Feld, Jordan J ; Pike, Kristen M ; Marciano, Beatriz E ; Uzel, Gulbu ; DeRavin, Suk See ; Priel, Debra A. Long ; Soule, Benjamin P ; Zarember, Kol A ; Malech, Harry L ; Holland, Steven M ; Gallin, John I
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2010-12, Vol.363 (27), p.2600-2610
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A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome
Author / Creator:McDermott, David H ; Velez, Daniel ; Cho, Elena ; Cowen, Edward W ; DiGiovanna, John J ; Pastrana, Diana V ; Buck, Christopher B ; Calvo, Katherine R ; Gardner, Pamela J ; Rosenzweig, Sergio D ; Stratton, Pamela ; Merideth, Melissa A ; Kim, H Jeffrey ; Brewer, Carmen ; Katz, James D ; Kuhns, Douglas B ; Malech, Harry L ; Follmann, Dean ; Fay, Michael P ; Murphy, Philip M
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2023-10, Vol.133 (19), p.1-16
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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Author / Creator:Sweeney, Colin L. ; Pavel-Dinu, Mara ; Choi, Uimook ; Brault, Julie ; Liu, Taylor ; Koontz, Sherry ; Li, Linhong ; Theobald, Narda ; Lee, Janet ; Bello, Ezekiel A. ; Wu, Xiaolin ; Meis, Ronald J. ; Dahl, Gary A. ; Porteus, Matthew H. ; Malech, Harry L. ; De Ravin, Suk See
Publisher:London: Nature Publishing Group UK
Journal title:Gene therapy, 2021-06, Vol.28 (6), p.373-390
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Homozygous IL37 mutation associated with infantile inflammatory bowel disease
Author / Creator:Zhang, Zinan Z. ; Zhang, Yu ; He, Tingyan ; Sweeney, Colin L. ; Barisc, Safa ; Karakoc-Aydiner, Elif ; Yao, Yikun ; Ertem, Deniz ; Matthews, Helen F. ; Gonzaga-Jauregui, Claudia ; Malech, Harry L. ; Su, Helen C. ; Ozen, Ahmet ; Smith, Kenneth G. C. ; Lenardo, Michael J.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2021-03, Vol.118 (10), p.1-3
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Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System
Author / Creator:Gardner, Cameron L. ; Pavel-Dinu, Mara ; Dobbs, Kerry ; Bosticardo, Marita ; Reardon, Paul K. ; Lack, Justin ; DeRavin, Suk See ; Le, Kent ; Bello, Ezekiel ; Pala, Francesca ; Delmonte, Ottavia M. ; Malech, Harry ; Montel-Hagan, Amelie ; Crooks, Gay ; Acuto, Oreste ; Porteus, Matthew H. ; Notarangelo, Luigi D.
Publisher:New York: Springer US
Journal title:Journal of clinical immunology, 2021-07, Vol.41 (5), p.852-862
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STAT3 Mutations in the Hyper-IgE Syndrome
Author / Creator:Holland, Steven M ; DeLeo, Frank R ; Elloumi, Houda Z ; Hsu, Amy P ; Uzel, Gulbu ; Brodsky, Nina ; Freeman, Alexandra F ; Demidowich, Andrew ; Davis, Joie ; Turner, Maria L ; Anderson, Victoria L ; Darnell, Dirk N ; Welch, Pamela A ; Kuhns, Douglas B ; Frucht, David M ; Malech, Harry L ; Gallin, John I ; Kobayashi, Scott D ; Whitney, Adeline R ; Voyich, Jovanka M ; Musser, James M ; Woellner, Cristina ; Schäffer, Alejandro A ; Puck, Jennifer M ; Grimbacher, Bodo
Publisher:Boston, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2007-10, Vol.357 (16), p.1608-1619
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Failure to Prevent Severe Graft-Versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in Chronic Granulomatous Disease
Author / Creator:Parta, Mark ; Hilligoss, Dianne ; Kelly, Corin ; Kwatemaa, Nana ; Theobald, Narda ; Zerbe, Christa S. ; Holland, Steven M. ; Malech, Harry L. ; Kang, Elizabeth M.
Publisher:New York: Springer US
Journal title:Journal of clinical immunology, 2020-05, Vol.40 (4), p.619-624
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Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction
Author / Creator:Sweeney, Colin L. ; Zou, Jizhong ; Choi, Uimook ; Merling, Randall K. ; Liu, Alexander ; Bodansky, Aaron ; Burkett, Sandra ; Kim, Jung-Woong ; De Ravin, Suk See ; Malech, Harry L.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2017-02, Vol.25 (2), p.321-330
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Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers
Author / Creator:Tsilifis, Christo ; Torppa, Tuulia ; Williams, Eleri J. ; Albert, Michael H. ; Hauck, Fabian ; Soncini, Elena ; Kang, Elizabeth ; Malech, Harry ; Schuetz, Catharina ; von Bernuth, Horst ; Slatter, Mary A. ; Gennery, Andrew R.
Publisher:New York: Springer US
Journal title:Journal of clinical immunology, 2023-11, Vol.43 (8), p.1964-1973
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PSG9 Stimulates Increase in FoxP3+ Regulatory T-Cells through the TGF-β1 Pathway
Author / Creator:Jones, Karlie ; Ballesteros, Angela ; Mentink-Kane, Margaret ; Warren, James ; Rattila, Shemona ; Malech, Harry ; Kang, Elizabeth ; Dveksler, Gabriela
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-07, Vol.11 (7), p.e0158050-e0158050
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Malech, Harry Lewis

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Malech, Harry Lewis

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Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle

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Neutrophil extracellular traps enriched in oxidized mitochondrial DNA are interferogenic and contribute to lupus-like disease

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Differentiation of human and murine induced pluripotent stem cells to microglia-like cells

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Gene correction for SCID-X1 in long-term hematopoietic stem cells

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Lentiviral Gene Therapy for Artemis-Deficient SCID

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Pyocyanin-enhanced neutrophil extracellular trap formation requires the NADPH oxidase

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Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

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Residual NADPH Oxidase and Survival in Chronic Granulomatous Disease

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A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome

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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

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Homozygous IL37 mutation associated with infantile inflammatory bowel disease

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Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System

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STAT3 Mutations in the Hyper-IgE Syndrome

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Failure to Prevent Severe Graft-Versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in Chronic Granulomatous Disease

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Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction

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Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers

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PSG9 Stimulates Increase in FoxP3+ Regulatory T-Cells through the TGF-β1 Pathway

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