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Harry L. Malech
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Journal Articles

Genetically corrected RAG2 -SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

Mara Pavel-Dinu, Cameron L. Gardner, Yusuke Nakauchi, Tomoki Kawai, Ottavia M. Delmonte, Boaz Palterer, Marita Bosticardo, Francesca Pala, Sebastien Viel, Harry L. Malech, Hana Y. Ghanim, Nicole M. Bode, Gavin L. Kurgan, Angela M. Detweiler, Christopher A. Vakulskas, Norma F. Neff, Adam Sheikali, Sherah T. Menezes, Jade Chrobok, Elaine M. Hernández González, Ravindra Majeti, Luigi D. Notarangelo, Matthew H. Porteus
Journal: Blood Advances
Blood Adv (2024) 8 (7): 1820–1833.
https://doi.org/10.1182/bloodadvances.2023011766
Published: 2024
Includes: Supplemental data
Journal Articles

NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease

Suk See De Ravin, Julie Brault, Ronald J. Meis, Linhong Li, Narda Theobald, Aylin C. Bonifacino, Hong Lei, Taylor Q. Liu, Sherry Koontz, Cristina Corsino, Marissa A. Zarakas, Jigar V. Desai, Aaron B. Clark, Uimook Choi, Mark E. Metzger, Kamille West, Steven L. Highfill, Elizabeth Kang, Douglas B. Kuhns, Michail S. Lionakis, David F. Stroncek, Cynthia E. Dunbar, John F. Tisdale, Robert E. Donahue, Gary A. Dahl, Harry L. Malech
Journal: Blood Advances
Blood Adv (2020) 4 (23): 5976–5987.
https://doi.org/10.1182/bloodadvances.2020003224
Published: 2020
Journal Articles

Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia

Marita Bosticardo, Francesca Pala, Enrica Calzoni, Ottavia M. Delmonte, Kerry Dobbs, Cameron L. Gardner, Nicolo’ Sacchetti, Tomoki Kawai, Elizabeth K. Garabedian, Debbie Draper, Jenna R. E. Bergerson, Suk See DeRavin, Alexandra F. Freeman, Tayfun Güngör, Nicholas Hartog, Steven M. Holland, Donald B. Kohn, Harry L. Malech, Mary Louise Markert, Katja G. Weinacht, Anna Villa, Christopher S. Seet, Amelie Montel-Hagen, Gay M. Crooks, Luigi D. Notarangelo
Journal: Blood Advances
Blood Adv (2020) 4 (12): 2611–2616.
https://doi.org/10.1182/bloodadvances.2020001730
Published: 2020
Includes: Supplemental data
Journal Articles

NCF1 (p47 phox )–deficient chronic granulomatous disease: comprehensive genetic and flow cytometric analysis

Douglas B. Kuhns, Amy P. Hsu, David Sun, Karen Lau, Danielle Fink, Paul Griffith, Da Wei Huang, Debra A. Long Priel, Laura Mendez, Samantha Kreuzburg, Christa S. Zerbe, Suk See De Ravin, Harry L. Malech, Steven M. Holland, Xiaolin Wu, John I. Gallin
Journal: Blood Advances
Blood Adv (2019) 3 (2): 136–147.
https://doi.org/10.1182/bloodadvances.2018023184
Published: 2019
Includes: Supplemental data
Journal Articles

Gene-edited pseudogene resurrection corrects p47 phox -deficient chronic granulomatous disease

Randall K. Merling, Douglas B. Kuhns, Colin L. Sweeney, Xiaolin Wu, Sandra Burkett, Jessica Chu, Janet Lee, Sherry Koontz, Giovanni Di Pasquale, Sandra A. Afione, John A. Chiorini, Elizabeth M. Kang, Uimook Choi, Suk See De Ravin, Harry L. Malech
Journal: Blood Advances
Blood Adv (2016) 1 (4): 270–278.
https://doi.org/10.1182/bloodadvances.2016001214
Published: 2016
Includes: Supplemental data