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Journal Articles

Clinical and functional spectrum of RAC2-related immunodeficiency

Clinical Trials & Observations
Ágnes Donkó, Svetlana O. Sharapova, Juraj Kabat, Sundar Ganesan, Fabian H. Hauck, Jenna R. E. Bergerson, Louis Marois, Jordan Abbott, Despina Moshous, Kelli W. Williams, Nicholas Campbell, Paul L. Martin, Chantal Lagresle-Peyrou, Timothy Trojan, Natalia B. Kuzmenko, Ekaterina A. Deordieva, Elena V. Raykina, Michael S. Abers, Hassan Abolhassani, Vincent Barlogis, Carlos Milla, Geoffrey Hall, Talal Mousallem, Joseph Church, Neena Kapoor, Guilhem Cros, Hugo Chapdelaine, Clara Franco-Jarava, Ingrid Lopez-Lerma, Maurizio Miano, Jennifer W. Leiding, Christoph Klein, Marie José Stasia, Alain Fischer, Kuang-Chih Hsiao, Timi Martelius, Mikko R. J. Sepännen, Sara Barmettler, Jolan Walter, Tania N. Masmas, Anna A. Mukhina, Emilia Liana Falcone, Sven Kracker, Anna Shcherbina, Steven M. Holland, Thomas L. Leto, Amy P. Hsu
Journal: Blood
Blood (2024) 143 (15): 1476–1487.
https://doi.org/10.1182/blood.2023022098
Published: 2024
Includes: Supplemental data
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

Ewelina Mamcarz, MD, Sheng Zhou, PhD, Timothy Lockey, PhD, Shannon Boi, PhD, Yan Koon-Kiu, PhD, Shane Cross, Pharm.D., Guolian Kang, PhD, Zhijun Ma, MD, Jose Marcos Condori, PhD, Jolanta Dowdy, M.S., Jean-Yves Metais, PhD, Deanna Langfitt, PhD, Brandon Triplett, MD, Chen Li, M.S., Xiwen Zhao, MSPH, Gabriela Maron, MD, Sneha Suresh, MD, Juan Carlos Aldave Becerra, MD, Joseph Church, MD, Elif Dokmeci, MD, James T Love, MD, PhD, Hedi van der Watt, MD, Ana C da Matta Ain, MD, Christa Krupski, DO, MPH, Jiyang Yu, PhD, Lance E. Palmer, PhD, William Janssen, PhD, Suk See De Ravin, MD, PhD, Mitchell J. Weiss, MD, PhD, Benjamin Youngblood, PhD, Janel R Long-Boyle, PharmD, PhD, Michael M Meagher, PhD, Harry L. Malech, MD, Jennifer Puck, MD, Morton J Cowan, MD, Stephen Gottschalk, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2058.
https://doi.org/10.1182/blood-2019-126746
Published: 2019
Journal Articles

A Clinical Trial of Retroviral-Mediated Transfer of a rev -Responsive Element Decoy Gene Into CD34 + Cells From the Bone Marrow of Human Immunodeficiency Virus-1–Infected Children

Donald B. Kohn, Gerhard Bauer, C. Robert Rice, J.C. Rothschild, Denise A. Carbonaro, Penelope Valdez, Qian-lin Hao, Chen Zhou, Ingrid Bahner, Karen Kearns, Kate Brody, Sarah Fox, Elizabeth Haden, Kathy Wilson, Cathy Salata, Cathy Dolan, Charles Wetter, Estuardo Aguilar-Cordova, Joseph Church
Journal: Blood
Blood (1999) 94 (1): 368–371.
https://doi.org/10.1182/blood.V94.1.368.413a47_368_371
Published: 1999