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Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Emmanuelle Six, Agathe Guilloux, Adeline Denis, Arnaud Lecoules, Alessandra Magnani, Romain Vilette, Frances Male, Nicolas Cagnard, Marianne Delville, Elisa Magrin, Laure Caccavelli, Cécile Roudaut, Clemence Plantier, Steicy Sobrino, John Gregg, Christopher L. Nobles, John K. Everett, Salima Hacein-Bey-Abina, Anne Galy, Alain Fischer, Adrian J. Thrasher, Isabelle André, Marina Cavazzana, Frederic D. Bushman
Journal: Blood
Blood (2020) 135 (15): 1219–1231.
https://doi.org/10.1182/blood.2019002350
Published: 2020
Includes: Supplemental data
Journal Articles

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Emmanuelle Six, Agathe Guilloux, Christopher Nobles, Rawya Zreik, Arnaud Lecoules, John Everett, Romain Vilette, Nicolas Cagnard, PhD, Frances Male, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Caccavelli, Cecile Roudaut, Clemence Plantier, Anne Galy, PharmD, PhD, Adrian J Thrasher, MD PhD FMedSci, Isabelle Andre-Schmutz, Charles C Berry, Marina Cavazzana, MD PhD, Frederic D Bushman, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4618.
https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618
Published: 2017
Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Clinical Trials & Observations
Emma C. Morris, Thomas Fox, Ronjon Chakraverty, Rita Tendeiro, Katie Snell, Christine Rivat, Sarah Grace, Kimberly Gilmour, Sarita Workman, Karen Buckland, Katie Butler, Ronnie Chee, Alan D. Salama, Hazem Ibrahim, Havinder Hara, Cecile Duret, Fulvio Mavilio, Frances Male, Frederick D. Bushman, Anne Galy, Siobhan O. Burns, H. Bobby Gaspar, Adrian J. Thrasher
Journal: Blood
Blood (2017) 130 (11): 1327–1335.
https://doi.org/10.1182/blood-2017-04-777136
Published: 2017
Includes: Supplemental data
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
Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Salima Hacein-Bey-Abina, Pharm.D., Ph.D., Sung-Yun Pai, MD, Frederic Bushman, PhD, Myriam Armant, PhD, Stephane Blanche, MD, Johanna Blondeau, Laure Caccavelli, Heather Daley, BS, Colleen Dansereau, RN, BSN, CPN, CPHON, Satiro deOliveira, MD, Dongjing Guo, MPH, Gregory Hopkins, BS, Annick Lim, M.Sci, Wendy B. London, PHD, Nirav Malani, Frances Male, BA, Punam Malik, MD, Maria Angelica Marinovic, MD, Sean McDonough, MS, Matias Oleastro, MD, Capucine Picard, MD PhD, Jerome Ritz, MD, Axel Schambach, MD PhD, Leslie E Silberstein, MD, Alla Tsytsykova, PhD, Johannes C.M. Van der Loo, PhD, Christopher Baum, MD, H. Bobby Gaspar, MD PhD, Donald B. Kohn, MD, Alexandra H. Filipovich, MD, Luigi Daniele Notarangelo, MD, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 715.
https://doi.org/10.1182/blood.V122.21.715.715
Published: 2013