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Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Clinical Trials & Observations
Roxane Labrosse, Julia I. Chu, Myriam A. Armant, John K. Everett, Danilo Pellin, Niharika Kareddy, Andrew L. Frelinger, Lauren A. Henderson, Amy E. O’Connell, Amlan Biswas, Jet Coenen-van der Spek, Alexandra Miggelbrink, Claudia Fiorini, Hriju Adhikari, Charles C. Berry, Vito Adrian Cantu, Johnson Fong, Jason Jaroslavsky, Derin F. Karadeniz, Quan-Zhen Li, Shantan Reddy, Aoife M. Roche, Chengsong Zhu, Jennifer S. Whangbo, Colleen Dansereau, Brenda Mackinnon, Emily Morris, Stephanie M. Koo, Wendy B. London, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Jenny M. Despotovic, Lisa R. Forbes Satter, Akihiko Saitoh, Yuta Aizawa, Alejandra King, Mai Anh Thi Nguyen, Vy Do Uyen Vu, Scott B. Snapper, Anne Galy, Luigi D. Notarangelo, Frederic D. Bushman, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2023) 142 (15): 1281–1296.
https://doi.org/10.1182/blood.2022019117
Published: 2023
Includes: Supplemental data
Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Roxane Labrosse, MD FRCPC, Julia Chu, MD MPH, Myriam Armant, PhD, Jet van der Spek, MD, Alexandra Miggelbrink, MD, Johnson Fong, John K. Everett, PhD, Hayley Raymond, BA, Lyanna Kessler, Colleen Dansereau, MSN, RN, CPN, Brenda Mackinnon, BSN, RN, Stephanie Koo, Emily Morris, Wendy B. London, PhD, Ahmet Ozen, MD, Safa Baris, MD, Jenny M. Despotovic, DO, MS, Lisa Forbes, MD, Akihiko Saitoh, MD, Takayuki Takachi, MD, Alejandra King, MD, Thi Mai Anh Thi Nguyen, MD, Vy Do Uyen Vu, MD, Frederic D. Bushman, PhD, Anne Galy, PharmD, PhD, Luigi Notarangelo, MD, David A Williams, MD, Sung-Yun Pai, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4629.
https://doi.org/10.1182/blood-2019-126161
Published: 2019
Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Julia I Chu, MD MPH, Lauren A Henderson, MD, Myriam Armant, PhD, Frances Male, BA, Colleen H Dansereau, MSN, RN, Brenda MacKinnon, RN, Christopher J Burke, Matthew E Cavanaugh, Wendy B. London, PhD, Isil B Barlan, MD, Ahmet Özen, MD, Safa Baris, MD, Elif Karakoc-Aydiner, MD, Ayça Kiykim, MD, Imelda Celine Hanson, MD, Jenny M. Despotovic, DO, MS, Akihiko Saitoh, MD PhD, Takayuki Takachi, MD PhD, Kohsuke Imai, MD PhD, Alejandra King, MD, Silvia Arredondo, MD, Jerome Ritz, MD, Frederic D Bushman, PhD, Anne Galy, PharmD, PhD, Luigi Daniele Notarangelo, MD, David A. Williams, MD, Sung-Yun Pai, MD
Journal: Blood
Blood (2015) 126 (23): 260.
https://doi.org/10.1182/blood.V126.23.260.260
Published: 2015