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Harry L. Malech

David Geffen School of Medicine at UCLA

96H-index
561Paper Count
3.4WCitation Count
Published Papers 181
Publication Date
Additive effects of common and rare genetic variants on inflammatory bowel disease risk in a cohort with immunodeficiency
err2026-02-27
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PREAI
errSruthi Srinivasan; Wenjia Cao; Morgan N. Similuk; Rajarshi Ghosh; Bryce A. Seifert; Mari Tokita; Justin Lack; Beatriz E. Marciano; Christa S. Zerbe; Pamela A. Frischmeyer-Guerrerio; Gulbu Uzel; Ivan Fuss; Alexandra F. Freeman; Michail S. Lionakis; Luigi D. Notarangelo; Ottavia M. Delmonte; Harry L. Malech; Jenna R.E. Bergerson; Steven M. Holland; Magdalena A. Walkiewicz
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Prime Editing for p47phox-Deficient Chronic Granulomatous Disease
err2025-12-01
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PREAI
errGori, Jennifer L.; Haddad, Elie; Frangoul, Haydar; Kohn, Donald B.; Morris, Emma C.; Martin, Bradley N.; Deary, Briana A.; Nickerson, McKinley; Scholz, Rebecca L.; Fernandez, Isabel; Leveille, Karine; De Ravin, Suk See; Kang, Elizabeth M.; Pierzynski, Marie; Estwick, Tyra; Littel, Patricia; Kuhns, Douglas B.; Long Priel, Debra A.; Teira, Pierre; Turvey, Stuart; Arnold, Jordyn; Evans, Misty D.; McManus, Meghann; Carpenter, Ben; Waterman, David P.; Anzalone, Andrew V.; Petrusich, Alexandria; Osuna, Christa E.; O'Malley, Tiernan T.; Stewart-Ornstein, Jacob; Heath, Jack M.; Nehilla, Barrett J.; Asmal, Mohammed; Malech, Harry L.
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Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
err2025-10-16
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PREAI
errBooth, Claire; Masiuk, Katelyn; Vazouras, Konstantinos; Fernandes, Augustine; Xu-Bayford, Jinhua; Campo Fernandez, Beatriz; Roy, Sohini; Curio-Penny, Beatrice; Arnold, Jordyn; Terrazas, Dayna; Reid, Jack; Gilmour, Kimberly C.; Adams, Stuart; Alvarez Mediavilla, Elena; Mhaldien, Lana; O'Toole, Grainne; Ahmed, Rima; Garabedian, Elizabeth; Malech, Harry; De Ravin, Suk See; Moore, Theodore B.; De Oliveira, Satiro; Pellin, Danilo; Lin, Tsai-Yu; Dang, Thao Thi; Cornetta, Kenneth; Hershfield, Michael S.; Hara, Havinder; Thrasher, Adrian J.; Gaspar, H. Bobby; Kohn, Donald B.
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Umbilical Cord Blood Transplantation Provides an Alternative for Patients with Chronic Granulomatous Disease Lacking HLA-Matched Donors: a PIDTC Report
err2025-10-02
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PREAI
errDanielle E. Arnold; Jennifer W. Leiding; Brent Logan; Rebecca A. Marsh; Linda M. Griffith; Eyal Grunebaum; Luis Murguía-Favela; Kanwaldeep Mallhi; Deepakbabu Chellapandian; Christin L. Deal; Stephanie Si Lim; Vinod Prasad; Jennifer Heimall; Shanmuganathan Chandrakasan; Karin Chen; Lolie C. Yu; Christine M. Seroogy; Alfred Gillio; Jeffrey J. Bednarski; Neena Kapoor; Theodore B. Moore; Geoffrey D.E. Cuvelier; Fabien Touzot; Ahmad Rayes; Christen L. Ebens; Edo Schaefer; Andrea Bauchat; Ashley Chopek; Lauri Burroughs; Morton J. Cowan; Christopher C. Dvorak; Elie Haddad; Donald B. Kohn; Luigi D. Notarangelo; Sung-Yun Pai; Jennifer M. Puck; Michael A. Pulsipher; Troy Torgerson; Harry L. Malech; Elizabeth M. Kang; Suhag Parikh
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Reliable genetic diagnosis of NCF1 (p47phox)-deficient chronic granulomatous disease using high-throughput sequencing
err2025-08-18
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errOAAI
errAmy P. Hsu; Eric Karlins; Justin Lack; T. Joseph Pepper; Karen Lau; Kimberly R. Marshall-Batty; Debra Long Priel; Joie Davis; Danielle L. Fink; Christa S. Zerbe; John I. Gallin; Harry L. Malech; Steven M. Holland; Douglas B. Kuhns
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Usefulness and Limitations of Polymerase Chain Reaction (PCR) for the Diagnosis and Management of Toxoplasmosis Following Allogeneic Hematopoietic Cell Transplant: Single-center Experience With 31 Patients Over 16 Years
err2025-08-05
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errOAAI
errMary M Czech; Theresa Jerussi; Sanchita Das; Rose Lee; Christopher G Kanakry; Jennifer Kanakry; Dimana Dimitrova; Mustafa A Hyder; Kamil Rechache; Winnie Trang; Daniel H Fowler; Michael R Bishop; Richard W Childs; Georg Aue; John Tisdale; Matthew Hsieh; Courtney Fitzhugh; Emily Limerick; Dennis Hickstein; Harry L Malech; Elizabeth M Kang; Steven Pavletic; Danielle E Arnold; Sung-Yun Pai; Jennifer Cuellar-Rodriguez; Juan C Gea-Banacloche
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Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable
err2025-07-15
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PREAI
errSadik H. Kassim; Fyodor Urnov; Kiran Musunuru; Ann Lee; Luis Barrera; Pamela Stetkiewicz; Julianne Bruno; Matthew Hewitt; Troy Lister; Harry Malech; Lindsay Gasch; Matt Diver; Nicholas Gertler; Felix Grignon; Audrey Le; Michael Lehmicke; Edwin M. Horwitz; David R. Liu; Josephine Lembong; Vanessa Almendro-Navarro
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Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher
err2025-06-24
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PREAI
errFyodor Urnov; Sadik Kassim; Kiran Musunuru; David Liu; Ann Lee; Luis Barrera; Pam Stetkiewicz; Julianne Bruno; Matthew Hewitt; Troy Lister; Harry Malech; Lindsay Gasch; Matt Diver; Nicholas Gertler; Felix Grignon; Audrey Le; Michael Lehmicke; Vanessa Almendro-Navarro; Josephine Lembong
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High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease
err2024-10-16
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PREAI
errBzhilyanskaya, Vera; Ma, Linyuan; Liu, Siyuan; Fox, Lauren R.; Whittaker, Madelynn N.; Meis, Ronald J.; Choi, Uimook; Lawson, Amanda; Ma, Michelle; Theobald, Narda; Burkett, Sandra; Sweeney, Colin L.; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Lack, Justin B.; Wu, Xiaolin; Dahl, Gary A.; Malech, Harry L.; Kleinstiver, Benjamin P.; De Ravin, Suk See
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Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study
err2024-05-01
err3
PREAI
errGrunebaum, Eyal; Arnold, Danielle E.; Logan, Brent; Parikh, Suhag; Marsh, Rebecca A.; Griffith, Linda M.; Mallhi, Kanwaldeep; Chellapandian, Deepak; Lim, Stephanie Si; Deal, Christin L.; Kapoor, Neena; Murguia-Favela, Luis; Falcone, Emilia Liana; Prasad, Vinod K.; Touzot, Fabien; Bleesing, Jack J.; Chandrakasan, Shanmuganathan; Heimall, Jennifer R.; Bednarski, Jeffrey J.; Broglie, Larisa A.; Chong, Hey Jin; Kapadia, Malika; Prockop, Susan; Saldana, Blachy J. Davila; Schaefer, Edo; Bauchat, Andrea L.; Teira, Pierre; Chandra, Sharat; Parta, Mark; Cowan, Morton J.; Dvorak, Christopher C.; Haddad, Elie; Kohn, Donald B.; Notarangelo, Luigi D.; Pai, Sung-Yun; Puck, Jennifer M.; Pulsipher, Michael A.; Torgerson, Troy R.; Malech, Harry L.; Kang, Elizabeth M.; Leiding, Jennifer W.
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Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
err2024-04-09
err1
errOAAI
errPavel-Dinu, Mara; Gardner, Cameron L.; Nakauchi, Yusuke; Kawai, Tomoki; Delmonte, Ottavia M.; Palterer, Boaz; Bosticardo, Marita; Pala, Francesca; Viel, Sebastien; Malech, Harry L.; Ghanim, Hana Y.; Bode, Nicole M.; Kurgan, Gavin L.; Detweiler, Angela M.; Vakulskas, Christopher A.; Neff, Norma F.; Sheikali, Adam; Menezes, Sherah T.; Chrobok, Jade; Gonzalez, Elaine M. Hernandez; Majeti, Ravindra; Notarangelo, Luigi D.; Porteus, Matthew H.
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Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
err2024-02-07
err5
errOAAI
errCastiello, Maria Carmina; Brandas, Chiara; Ferrari, Samuele; Porcellini, Simona; Sacchetti, Nicolo; Canarutto, Daniele; Draghici, Elena; Merelli, Ivan; Barcella, Matteo; Pelosi, Gabriele; Vavassori, Valentina; Varesi, Angelica; Jacob, Aurelien; Scala, Serena; Basso Ricci, Luca; Paulis, Marianna; Strina, Dario; Di Verniere, Martina; Sergi Sergi, Lucia; Serafini, Marta; Holland, Steven M.; Bergerson, Jenna R. E.; De Ravin, Suk See; Malech, Harry L.; Pala, Francesca; Bosticardo, Marita; Brombin, Chiara; Cugnata, Federica; Calzoni, Enrica; Crooks, Gay M.; Notarangelo, Luigi D.; Genovese, Pietro; Naldini, Luigi; Villa, Anna
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Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study
err2024-01-01
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errOAAI
errEissa, Hesham; Thakar, Monica S.; Shah, Ami J.; Logan, Brent R.; Griffith, Linda M.; Dong, Huaying; Parrott, Roberta E.; O'Reilly, Richard J.; Dara, Jasmeen; Kapoor, Neena; Satter, Lisa Forbes; Chandra, Sharat; Kapadia, Malika; Chandrakasan, Shanmuganathan; Knutsen, Alan; Jyonouchi, Soma C.; Molinari, Lyndsay; Rayes, Ahmad; Ebens, Christen L.; Teira, Pierre; Saldana, Blachy J. Davila; Burroughs, Lauri M.; Chaudhury, Sonali; Chellapandian, Deepak; Gillio, Alfred P.; Goldman, Fredrick; Malech, Harry L.; DeSantes, Kenneth; Cuvelier, Geoff D. E.; Rozmus, Jacob; Quinones, Ralph; Yu, Lolie C.; Broglie, Larisa; Aquino, Victor; Shereck, Evan; Moore, Theodore B.; Vander Lugt, Mark T.; Mousallem, Talal I.; Oved, Joeseph H.; Dorsey, Morna; Abdel-Azim, Hisham; Martinez, Caridad; Bleesing, Jacob H.; Prockop, Susan; Kohn, Donald B.; Bednarski, Jeffrey J.; Leiding, Jennifer; Marsh, Rebecca A.; Torgerson, Troy; Notarangelo, Luigi D.; Pai, Sung-Yun; Pulsipher, Michael A.; Puck, Jennifer M.; Dvorak, Christopher C.; Haddad, Elie; Buckley, Rebecca H.; Cowan, Morton J.; Heimall, Jennifer
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Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease
err2023-12-01
err8
errOAAI
errChandrasekaran, Prabha; Han, Yu; Zerbe, Christa S.; Heller, Theo; DeRavin, Suk See; Kreuzberg, Samantha A.; Marciano, Beatriz E.; Siu, Yik; Jones, Drew R.; Abraham, Roshini S.; Stephens, Michael C.; Tsou, Amy M.; Snapper, Scott; Conlan, Sean; Subramanian, Poorani; Quinones, Mariam; Grou, Caroline; Calderon, Virginie; Deming, Clayton; Leiding, Jennifer W.; Arnold, Danielle E.; Logan, Brent R.; Griffith, Linda M.; Petrovic, Aleksandra; Mousallem, Talal I.; Kapoor, Neena; Heimall, Jennifer R.; Barnum, Jessie L.; Kapadia, Malika; Wright, Nicola; Rayes, Ahmad; Chandra, Sharat; Broglie, Larisa A.; Chellapandian, Deepak; Deal, Christin L.; Grunebaum, Eyal; Lim, Stephanie Si; Mallhi, Kanwaldeep; Marsh, Rebecca A.; Murguia-Favela, Luis; Parikh, Suhag; Touzot, Fabien; Cowan, Morton J.; Dvorak, Christopher C.; Haddad, Elie; Kohn, Donald B.; Notarangelo, Luigi D.; Pai, Sung-Yun; Puck, Jennifer M.; Pulsipher, Michael A.; Torgerson, Troy R.; Kang, Elizabeth M.; Malech, Harry L.; Segre, Julia A.; Bryant, Clare E.; Holland, Steven M.; Falcone, Emilia Liana
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A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome
err2023-10-02
err12
errOAAI
errMcdermott, 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.
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Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease-associated inflammatory bowel disease
err2023-09-24
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errOAAI
errBhattacharya, Sumona; Wong, Uni; Khalid, Mian; Blaney, Hanna; Menkart, Matthew G.; Oringher, Jenna L.; Taneja, Sonia L.; Zerbe, Christa S.; De Ravin, Suk See; Malech, Harry L.; Holland, Steven M.; Koh, Christopher; Heller, Theo
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Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers
err2023-08-24
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errOAAI
errTsilifis, 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.
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Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients
err2023-06-01
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errOAAI
errRiller, Quentin; Fourgeaud, Jacques; Bruneau, Julie; De Ravin, Suk See; Smith, Grace; Fusaro, Mathieu; Meriem, Samy; Magerus, Aude; Luka, Marine; Abdessalem, Ghaith; Lhermitte, Ludovic; Jamet, Anne; Six, Emmanuelle; Magnani, Alessandra; Castelle, Martin; Levy, Romain; Lecuit, Mathilde M.; Fournier, Benjamin; Winter, Sarah; Semeraro, Michaela; Pinto, Graziella; Abid, Hanene; Mahlaoui, Nizar; Cheikh, Nathalie; Florkin, Benoit; Frange, Pierre; Jeziorski, Eric; Suarez, Felipe; Sarrot-Reynauld, Francoise; Nouar, Dalila; Debray, Dominique; Lacaille, Florence; Picard, Capucine; Perot, Philippe; Regnault, Beatrice; Da Rocha, Nicolas; de Cevins, Camille; Delage, Laure; Perot, Brieuc P.; Vinit, Angelique; Carbone, Francesco; Brunaud, Camille; Marchais, Manon; Stolzenberg, Marie-Claude; Asnafi, Vahid; Molina, Thierry; Rieux-Laucat, Frederic; Notarangelo, Luigi D.; Pittaluga, Stefania; Philippe, Jean; Moshous, Despina; Blanche, Stephane; Malech, Harry; Eloit, Marc; Cavazzana, Marina; Fischer, Alain; Menager, Mickaeeurol M.; Neven, Benedicte
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Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease
err2023-05-01
err5
errOAAI
errYang, Alexander H.; Sullivan, Brigit; Zerbe, Christa S.; De Ravin, Suk See; Blakely, Andrew M.; Quezado, Martha M.; Marciano, Beatriz E.; Marko, Jamie; Ling, Alexander; Kleiner, David E.; Gallin, John I.; Malech, Harry L.; Holland, Steven M.; Heller, Theo
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Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells
err2023-04-01
err5
errOAAI
errTamaoki, Naritaka; Siebert, Stefan; Maeda, Takuya; Ha, Ngoc-Han; Good, Meghan L.; Huang, Yin; Vodnala, Suman K.; Haro-Mora, Juan J.; Uchida, Naoya; Tisdale, John F.; Sweeney, Colin L.; Choi, Uimook; Brault, Julie; Koontz, Sherry; Malech, Harry L.; Yamazaki, Yasuhiro; Isonaka, Risa; Goldstein, David S.; Kimura, Masaki; Takebe, Takanori; Zou, Jizhong; Stroncek, David F.; Robey, Pamela G.; Kruhlak, Michael J.; Restifo, Nicholas P.; Vizcardo, Raul
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