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Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 631.
https://doi.org/10.1182/blood-2018-99-118050
Published: 2018
Journal Articles

Long Term Follow up after Adoptive Transfer of CD19-Specific CAR + T Cells Genetically Modified Via Non-Viral Sleeping Beauty S ystem Following Hematopoietic Stem Cell Transplantation (HSCT)

Partow Kebriaei, MD, Helen Huls, Simon Olivares, Aaron F Orozco, Shihuang Su, Sourindra N Maiti, Elenanor De Groot, Amy Smith, Yijun Yang, PhD, Gabriela Rondon, MD, Muzaffar H. Qazilbash, MD, Stefan O. Ciurea, Amin M. Alousi, Yago Nieto, MD PhD, David Costa Marin, MD, Uday Popat, MD, Chitra Hosing, MD, Hagop M. Kantarjian, MD, Katayoun Rezvani, MD PhD, Elizabeth J Shpall, MD, Richard E Champlin, MD, Laurence J N Cooper, MDPhD, Harjeet Singh, MSc, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 2059.
https://doi.org/10.1182/blood.V130.Suppl_1.2059.2059
Published: 2017
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
Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Sung-Yun Pai, MD, Luigi Daniele Notarangelo, MD, Chad Harris, Federica Cattaneo, MD, Matthew Wladkowski, Myriam Armant, PhD, Grace S. Kao, MD, Matthew Wielgosz, Arthur W. Nienhuis, MD, Ute Modlich, DVM, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Michael Rothe, Susannah I Thornhill, PhD, H. Bobby Gaspar, Kathryn Parsley, PhD, Samantha Cooray, PhD, Elke Grassman, Ph.D., Frederic Bushman, PhD, Sally Shupien, Matias Oleastro, MD, Salima Hacein-Bey-Abina, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, Punam Malik, MD, Alexandra Filipovich, MD, Donald B. Kohn, MD, Christopher Baum, MD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2011) 118 (21): 164.
https://doi.org/10.1182/blood.V118.21.164.164
Published: 2011
Journal Articles

Novel Gene Delivery System of Sendai Virus Transduced Human Peripheral Monocytes.

Tomoko Tanaka, Ryo Kurita, Ph.D., Kashiya Takasugi, M.D., Takafumi Nakamura, Ph.D., Makoto Inoue, Ph.D., Mamoru Hasegawa, Ph.D., Fumiko Ono, Ph.D., Keiji Terao, Ph.D., Yasuhiro Yoshikawa, Ph.D., Kenzaburo Tani, M.D.,Ph.D.
Journal: Blood
Blood (2006) 108 (11): 5482.
https://doi.org/10.1182/blood.V108.11.5482.5482
Published: 2006
Journal Articles

Efficient Induction of Tolerance to FIX by Direct Intramuscular Delivery of AAV1.

Ellen F. Cohn, PhD, Meagan E. Kelly, BS, Jiacai Zhuo, MD, Hengjun Chao, MD
Journal: Blood
Blood (2006) 108 (11): 3287.
https://doi.org/10.1182/blood.V108.11.3287.3287
Published: 2006
Journal Articles

Hematopoietic Repopulation, In Vivo, with Genetically Defined Clones Derived from HOXB4 Expressing ES-Cells.

Sandra Pilat, PhD, Sebastian Carotta, PhD, Bernhard Schiedlmeier, PhD, Kenji Kamino, MD, Andreas Mairhofer, MS, Manfred Schmidt, PhD, Christoph von Kalle, MD, Peter Steinlein, PhD, Wolfram Ostertag, PhD, Christopher Baum, MD, Hartmut Beug, PhD, Hannes Klump, PhD
Journal: Blood
Blood (2005) 106 (11): 196.
https://doi.org/10.1182/blood.V106.11.196.196
Published: 2005
Journal Articles

HOXB4 Enforces Equivalent Fates of ES-Cell-Derived and Adult Hematopoietic Cells.

Sandra Pilat, Carotta Sebastian, PhD, Schiedlmeier Bernhard, PhD, Modlich Ute, PhD, Kamino Kenji, MD, Will Elke, PhD, Mairhofer Andreas, Steinlein Peter, PhD, Ostertag Wolfram, PhD, Beug Hartmut, PhD, Baum Christopher, MD, Klump Hannes, PhD
Journal: Blood
Blood (2004) 104 (11): 494.
https://doi.org/10.1182/blood.V104.11.494.494
Published: 2004
Journal Articles

Correction of Uroporphyrinogen Decarboxylase Deficiency (Hepatoerythropoietic Porphyria) in Epstein-Barr Virus-Transformed B-Cell Lines by Retrovirus-Mediated Gene Transfer: Fluorescence-Based Selection of Transduced Cells

Antonio Fontanellas, Frédéric Mazurier, François Moreau-Gaudry, Francis Belloc, Cécile Ged, Hubert de Verneuil
Journal: Blood
Blood (1999) 94 (2): 465–474.
https://doi.org/10.1182/blood.V94.2.465
Published: 1999