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Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Clinical Trials & Observations
Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek
Journal: Blood
Blood (2018) 131 (9): 1022–1031.
https://doi.org/10.1182/blood-2017-09-804419
Published: 2018
Includes: Supplemental data
Journal Articles

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Frank W.G. Leebeek, MD PhD, Marco Tangelder, MD PhD, Karina Meijer, MD PhD, Giancarlo Castaman, Federica Cattaneo, MD, Michiel Coppens, MD PhD, Peter Kampmann, MD, Robert Klamroth, Roger E.G. Schutgens, MD PhD, Erhard Seifried, MD PhD, Joachim Schwaeble, MD, Halvard Boenig, MD PhD, Maaike Hendriks, Deya Corzo, MD FAAP, Wolfgang A. Miesbach, MD PhD
Journal: Blood
Blood (2016) 128 (22): 2314.
https://doi.org/10.1182/blood.V128.22.2314.2314
Published: 2016
Journal Articles

IL-21 is the primary common γ chain-binding cytokine required for human B-cell differentiation in vivo

Mike Recher, Lucinda J. Berglund, Danielle T. Avery, Morton J. Cowan, Andrew R. Gennery, Joanne Smart, Jane Peake, Melanie Wong, Sung-Yun Pai, Sachin Baxi, Jolan E. Walter, Umaimainthan Palendira, Gillian A. Tangye, Michael Rice, Shannon Brothers, Waleed Al-Herz, Hans Oettgen, Hermann Eibel, Jennifer M. Puck, Federica Cattaneo, John B. Ziegler, Silvia Giliani, Stuart G. Tangye, Luigi D. Notarangelo
Journal: Blood
Blood (2011) 118 (26): 6824–6835.
https://doi.org/10.1182/blood-2011-06-362533
Published: 2011
Includes: Supplemental data
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

Optimal Thalassemia Free Survival and Minimal Regimen Related Toxicity in 50 Consecutive Transplants of High Risk Beta Thalassemia Pediatric Patients Using Myelablative Therapy with Intravenous Busulphan.

Sarah Marktel, MD, Robert Chiesa, MD, Barbara Cappelli, MD, Tito Roccia, MD, Ilaria Frugnoli, MD, Anna Noè, MD, Erika Biral, MD, Roberto Crocchiolo, MD, Costanza Evangelio, MD, Marco Fossati, MD, Sara Napolitano, MD, Clara Soliman, Laura Cursi, MD, Federica Cattaneo, MD, Laura Zito, PD, Rossana Fiori, MD, Katharina Fleischhauer, MD, Roberto Miniero, MD, Fabio Ciceri, MD, Maria Grazia Roncarolo, MD
Journal: Blood
Blood (2008) 112 (11): 3266.
https://doi.org/10.1182/blood.V112.11.3266.3266
Published: 2008
Journal Articles

Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients

Barbara Cassani, Massimiliano Mirolo, Federica Cattaneo, Ulrike Benninghoff, Michael Hershfield, Filippo Carlucci, Antonella Tabucchi, Claudio Bordignon, Maria Grazia Roncarolo, Alessandro Aiuti
Journal: Blood
Blood (2008) 111 (8): 4209–4219.
https://doi.org/10.1182/blood-2007-05-092429
Published: 2008
Includes: Supplemental data
Journal Articles

Deoxyadenosine Inhibits T-Cell Activation in ADA-SCID Patients through a cAMP/PKA-Dependent Pathway.

Barbara Cassani, Massimiliano Mirolo, Federica Cattaneo, Ulrike Benninghoff, Filippo Carlucci, Antonella Tabucchi, Claudio Bordignon, Maria Grazia Roncarolo, Alessandro Aiuti
Journal: Blood
Blood (2006) 108 (11): 1250.
https://doi.org/10.1182/blood.V108.11.1250.1250
Published: 2006
Journal Articles

Long-Term Safety and Efficacy of Stem Cell Gene Therapy for ADA-SCID.

Alessandro Aiuti, Ulrike Benninghoff, Barbara Cassani, Federica Cattaneo, Luciano Callegaro, Grazia Andolfi, Massimiliano Mirolo, Samantha Scaramuzza, Sarah Marktel, Antonella Tabucchi, Filippo Carlucci, Martha Eibl, Memet Aker, Shimon Slavin, Fabio Ciceri, Roberto Miniero, Claudio Bordignon, Maria Grazia Roncarolo
Journal: Blood
Blood (2006) 108 (11): 200.
https://doi.org/10.1182/blood.V108.11.200.200
Published: 2006