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Journal Articles

Diagnostic assay to assist clinical decisions for unclassified severe combined immune deficiency

Panojot Bifsha, Jennifer W. Leiding, Sung-Yun Pai, Aurelien B. L. Colamartino, Nicholas Hartog, Joseph A. Church, Benjamin R. Oshrine, Jennifer M. Puck, M. Louise Markert, Elie Haddad
Journal: Blood Advances
Blood Adv (2020) 4 (12): 2606–2610.
https://doi.org/10.1182/bloodadvances.2020001736
Published: 2020
Includes: Supplemental data
Journal Articles

In vitro and in vivo efficacy of an anti-CD203c conjugated antibody (AGS-16C3F) in mouse models of advanced systemic mastocytosis

Yanyan Zhang, Ghaith Wedeh, Liang He, Monika Wittner, Florian Beghi, Viviane Baral, Jean-Marie Launay, Siham Bibi, Fernando Doñate, Hosein Kouros-Mehr, Michel Arock, Fawzia Louache
Journal: Blood Advances
Blood Adv (2019) 3 (4): 633–643.
https://doi.org/10.1182/bloodadvances.2018026179
Published: 2019
Includes: Supplemental data
Journal Articles

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

Ornellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, Ranjita Devi Moirangthem, Camille Lévy, Kuiying Ma, Caroline Costa, Didier Nègre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, François-Loïc Cosset, Isabelle André, Els Verhoeyen
Journal: Blood Advances
Blood Adv (2019) 3 (3): 461–475.
https://doi.org/10.1182/bloodadvances.2018027508
Published: 2019
Includes: Supplemental data
Journal Articles

Successful in utero stem cell transplantation in X-linked severe combined immunodeficiency

Alessandra Magnani, Jean-Marie Jouannic, Jérémie Rosain, Aurélie Gabrion, Fabien Touzot, Cécile Roudaut, Sven Kracker, Nizar Mahlaoui, Antoine Toubert, Emmanuel Clave, Elisabeth A. Macintyre, Isabelle Radford-Weiss, Marion Alcantara, Elisa Magrin, Brigitte Ternaux, Jennifer Nisoy, Laure Caccavelli, Anne-Marie Darras, Capucine Picard, Stéphane Blanche, Marina Cavazzana
Journal: Blood Advances
Blood Adv (2019) 3 (3): 237–241.
https://doi.org/10.1182/bloodadvances.2018023176
Published: 2019
Includes: Supplemental data
Journal Articles

Application of a radiosensitivity flow assay in a patient with DNA ligase 4 deficiency

David Buchbinder, Matthew J. Smith, Misako Kawahara, Morton J. Cowan, Jeffrey S. Buzby, Roshini S. Abraham
Journal: Blood Advances
Blood Adv (2018) 2 (15): 1828–1832.
https://doi.org/10.1182/bloodadvances.2018016113
Published: 2018
Includes: Supplemental data
Journal Articles

Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

Olivier Humbert, Frieda Chan, Yogendra S. Rajawat, Troy R. Torgerson, Christopher R. Burtner, Nicholas W. Hubbard, Daniel Humphrys, Zachary K. Norgaard, Patricia O’Donnell, Jennifer E. Adair, Grant D. Trobridge, Andrew M. Scharenberg, Peter J. Felsburg, David J. Rawlings, Hans-Peter Kiem
Journal: Blood Advances
Blood Adv (2018) 2 (9): 987–999.
https://doi.org/10.1182/bloodadvances.2018016451
Published: 2018
Includes: Supplemental data
Journal Articles

Cadherin 17 mutation associated with leaky severe combined immune deficiency is corrected by HSCT

Clinical Trials & Observations
Angela R. Smith, Ioanna A. Rota, Stefano Maio, Michel J. Massaad, Troy C. Lund, Luigi D. Notarangelo, Georg A. Holländer, Bruce R. Blazar
Journal: Blood Advances
Blood Adv (2017) 1 (23): 2083–2087.
https://doi.org/10.1182/bloodadvances.2017010926
Published: 2017
Journal Articles

Neurologic event–free survival demonstrates a benefit for SCID patients diagnosed by newborn screening

Clinical Trials & Observations
Christopher C. Dvorak, Jennifer M. Puck, Justin T. Wahlstrom, Morna Dorsey, Alexis Melton, Morton J. Cowan
Journal: Blood Advances
Blood Adv (2017) 1 (20): 1694–1698.
https://doi.org/10.1182/bloodadvances.2017010835
Published: 2017
Journal Articles

Engraftment of chronic myelomonocytic leukemia cells in immunocompromised mice supports disease dependency on cytokines

Yanyan Zhang, Liang He, Dorothée Selimoglu-Buet, Chloe Jego, Margot Morabito, Christophe Willekens, M'boyba Khadija Diop, Patrick Gonin, Valérie Lapierre, Nathalie Droin, Eric Solary, Fawzia Louache
Journal: Blood Advances
Blood Adv (2017) 1 (14): 972–979.
https://doi.org/10.1182/bloodadvances.2017004903
Published: 2017
Includes: Supplemental data