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Matias Oleastro
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Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
Journal Articles

Inherited human Apollo deficiency causes severe bone marrow failure and developmental defects

Laëtitia Kermasson, Dmitri Churikov, Aya Awad, Riham Smoom, Elodie Lainey, Fabien Touzot, Séverine Audebert-Bellanger, Sophie Haro, Lauréline Roger, Emilia Costa, Maload Mouf, Adriana Bottero, Matias Oleastro, Chrystelle Abdo, Jean-Pierre de Villartay, Vincent Géli, Yehuda Tzfati, Isabelle Callebaut, Silvia Danielian, Gabriela Soares, Caroline Kannengiesser, Patrick Revy
Journal: Blood
Blood (2022) 139 (16): 2427–2440.
https://doi.org/10.1182/blood.2021010791
Published: 2022
Includes: Supplemental data
Journal Articles

Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype

Clinical Trials & Observations
Julie Toubiana, Satoshi Okada, Julia Hiller, Matias Oleastro, Macarena Lagos Gomez, Juan Carlos Aldave Becerra, Marie Ouachée-Chardin, Fanny Fouyssac, Katta Mohan Girisha, Amos Etzioni, Joris Van Montfrans, Yildiz Camcioglu, Leigh Ann Kerns, Bernd Belohradsky, Stéphane Blanche, Aziz Bousfiha, Carlos Rodriguez-Gallego, Isabelle Meyts, Kai Kisand, Janine Reichenbach, Ellen D. Renner, Sergio Rosenzweig, Bodo Grimbacher, Frank L. van de Veerdonk, Claudia Traidl-Hoffmann, Capucine Picard, Laszlo Marodi, Tomohiro Morio, Masao Kobayashi, Desa Lilic, Joshua D. Milner, Steven Holland, Jean-Laurent Casanova, Anne Puel
Journal: Blood
Blood (2016) 127 (25): 3154–3164.
https://doi.org/10.1182/blood-2015-11-679902
Published: 2016
Includes: Supplemental data
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