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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

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MDPhD, Xiaolin Wu, Narda Theobald, Janet S Lee, John Gray, PhD, Imelda Celine Hanson, MD, Luigi Notarangelo, MDPhD, Brian P. Sorrentino, MD, Harry L. Malech, MD
Journal: Blood
Blood (2015) 126 (23): 261.
https://doi.org/10.1182/blood.V126.23.261.261
Published: 2015
Journal Articles

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Clinical Trials & Observations
Fabio Candotti, Kit L. Shaw, Linda Muul, Denise Carbonaro, Robert Sokolic, Christopher Choi, Shepherd H. Schurman, Elizabeth Garabedian, Chimene Kesserwan, G. Jayashree Jagadeesh, Pei-Yu Fu, Eric Gschweng, Aaron Cooper, John F. Tisdale, Kenneth I. Weinberg, Gay M. Crooks, Neena Kapoor, Ami Shah, Hisham Abdel-Azim, Xiao-Jin Yu, Monika Smogorzewska, Alan S. Wayne, Howard M. Rosenblatt, Carla M. Davis, Celine Hanson, Radha G. Rishi, Xiaoyan Wang, David Gjertson, Otto O. Yang, Arumugam Balamurugan, Gerhard Bauer, Joanna A. Ireland, Barbara C. Engel, Gregory M. Podsakoff, Michael S. Hershfield, R. Michael Blaese, Robertson Parkman, Donald B. Kohn
Journal: Blood
Blood (2012) 120 (18): 3635–3646.
https://doi.org/10.1182/blood-2012-02-400937
Published: 2012
Includes: Supplemental data
Journal Articles

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

Clinical Trials & Observations
Robert Sokolic, Irina Maric, Chimene Kesserwan, Elizabeth Garabedian, I. Celine Hanson, Margaret Dodds, Rebecca Buckley, Andrew C. Issekutz, Naynesh Kamani, Kit Shaw, Ben Tan, Pawan Bali, Michael S. Hershfield, Donald B. Kohn, Alan S. Wayne, Fabio Candotti
Journal: Blood
Blood (2011) 118 (10): 2688–2694.
https://doi.org/10.1182/blood-2011-01-329359
Published: 2011
Journal Articles

X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease

Clinical Trials & Observations
Claire Booth, Kimberly C. Gilmour, Paul Veys, Andrew R. Gennery, Mary A. Slatter, Helen Chapel, Paul T. Heath, Colin G. Steward, Owen Smith, Anna O'Meara, Hilary Kerrigan, Nizar Mahlaoui, Marina Cavazzana-Calvo, Alain Fischer, Despina Moshous, Stephane Blanche, Jana Pachlopnik Schmid, Sylvain Latour, Genevieve de Saint-Basile, Michael Albert, Gundula Notheis, Nikolaus Rieber, Brigitte Strahm, Henrike Ritterbusch, Arjan Lankester, Nico G. Hartwig, Isabelle Meyts, Alessandro Plebani, Annarosa Soresina, Andrea Finocchi, Claudio Pignata, Emilia Cirillo, Sonia Bonanomi, Christina Peters, Krzysztof Kalwak, Srdjan Pasic, Petr Sedlacek, Janez Jazbec, Hirokazu Kanegane, Kim E. Nichols, I. Celine Hanson, Neena Kapoor, Elie Haddad, Morton Cowan, Sharon Choo, Joanne Smart, Peter D. Arkwright, Hubert B. Gaspar
Journal: Blood
Blood (2011) 117 (1): 53–62.
https://doi.org/10.1182/blood-2010-06-284935
Published: 2011
Includes: Supplemental data