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Patricia Littel
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Journal Articles

First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101

Free
Elie Haddad, Haydar Frangoul, Donald Kohn, Emma Morris, Jennifer Gori, Bradley Martin, Briana Deary, McKinley Nickerson, Alexandria Petrusich, Pierre Teira, Karine Leveille, Isabel Fernandez, Stuart Turvey, Elizabeth Kang, Suk See De Ravin, Marie Pierzynski, Tyra Estwick-McCann, Patricia Littel, Douglas Kuhns, Debra Long-Priel, Meghann McManus, Misty Evans, Ben Carpenter, Jordyn Arnold, Barrett Nehilla, Jacob Stewart-Ornstein, Tiernan O'Malley, Jack Heath, Mohammed Asmal, Harry Malech
Journal: Blood
Blood (2025) 146 (Supplement 1): 4319.
https://doi.org/10.1182/blood-2025-4319
Published: 2025
Journal Articles

A phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixafor

Free
Clinical Trials & Observations
David H. McDermott, Qian Liu, Daniel Velez, Lizbeeth Lopez, Sandra Anaya-O’Brien, Jean Ulrick, Nana Kwatemaa, Judy Starling, Thomas A. Fleisher, Debra A. Long Priel, Melissa A. Merideth, Robert L. Giuntoli, Moses O. Evbuomwan, Patricia Littel, Martha M. Marquesen, Dianne Hilligoss, Rosamma DeCastro, George J. Grimes, Samuel T. Hwang, Stefania Pittaluga, Katherine R. Calvo, Pamela Stratton, Edward W. Cowen, Douglas B. Kuhns, Harry L. Malech, Philip M. Murphy
Journal: Blood
Blood (2014) 123 (15): 2308–2316.
https://doi.org/10.1182/blood-2013-09-527226
Published: 2014
Includes: Supplemental data
Journal Articles

The CXCR4 antagonist plerixafor corrects panleukopenia in patients with WHIM syndrome

Free
Clinical Trials & Observations
David H. McDermott, Qian Liu, Jean Ulrick, Nana Kwatemaa, Sandra Anaya-O'Brien, Scott R. Penzak, Joao Oliveira Filho, Debra A. Long Priel, Corin Kelly, Mary Garofalo, Patricia Littel, Martha M. Marquesen, Diane Hilligoss, Rosamma DeCastro, Thomas A. Fleisher, Douglas B. Kuhns, Harry L. Malech, Philip M. Murphy
Journal: Blood
Blood (2011) 118 (18): 4957–4962.
https://doi.org/10.1182/blood-2011-07-368084
Published: 2011
Includes: Supplemental data