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Journal Articles

Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions

Elena Almarza, PhD, Cristina Mesa-Núñez, PhD, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, PhD, M Luz Lozano, Paula Rio, PhD, Rosa M Yañez, PhD, Ken Law, PhD, Brian C Beard, PhD, Jonathan D Schwartz, MD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5751.
https://doi.org/10.1182/blood-2019-122878
Published: 2019
Journal Articles

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Elena Almarza, PhD, Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, Jonathan D Schwartz, MD, Juan A Bueren
Journal: Blood
Blood (2018) 132 (Supplement 1): 5798.
https://doi.org/10.1182/blood-2018-99-111136
Published: 2018
Journal Articles

Hematopoietic Stem Cell Transplantation in 65 Patients with Leukocyte Adhesion Deficiency Type I and Type III — A Retrospective Multi-Center Study

Shahrzad Bakhtiar, MD, Dirk-Jan Eikema, Henric-Jan Blok, Mouhab Ayas, MD, Franco Locatelli, MD PhD, Pietro Merli, MD, Amos Toren, MD PhD, Gal Goldstein, MD, Gerard Michel, MD, Gülyüz Öztürk, MD, Ansgar Schulz, MD, Carsten Heilmann, MDc, Robert Wynn, MD, Olga Aleinikova, MD PhD, Yves Bertrand, MD, Abdelghani Tbakhi, MD, Ardeshir Ghavamzadeh, MD, Paul Veys, MD PhD, Arjan Lankester, MD PhD, Peter Bader, MD PhD, Andrew Gennery, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3330.
https://doi.org/10.1182/blood.V130.Suppl_1.3330.3330
Published: 2017
Journal Articles

Lentiviral Vectors Incorporating Ubiquitous Chromatin Opening Element Driving Canine CD18 Expression

Everette J. R. Nelson, PhD, Laura M. Tuschong, B.S., Dennis D. Hickstein, MD
Journal: Blood
Blood (2016) 128 (22): 5890.
https://doi.org/10.1182/blood.V128.22.5890.5890
Published: 2016
Journal Articles

Polyclonality of Retroviral Insertion Sites Following Successful Retroviral-Mediated Gene Transfer of CD18 in Canine Leukocyte Adhesion Deficiency.

Mehreen Hai, PhD, Thomas R. Bauer, Ph.D., Laura M. Tuschong, B.S., Yuchen Gu, Ph.D., Robert A. Sokolic, M.D., Dennis D. Hickstein, M.D.
Journal: Blood
Blood (2006) 108 (11): 5479.
https://doi.org/10.1182/blood.V108.11.5479.5479
Published: 2006
Journal Articles

Somatic revertant mosaicism in a patient with leukocyte adhesion deficiency type 1

Clinical Trials & Observations Brief Report
Yumi Tone, Taizo Wada, Fumie Shibata, Tomoko Toma, Yoko Hashida, Yoshihito Kasahara, Shoichi Koizumi, Akihiro Yachie
Journal: Blood
Blood (2006) 109 (3): 1182–1184.
https://doi.org/10.1182/blood-2006-08-039057
Published: 2006
Journal Articles

Unique CD18 mutations involving a deletion in the extracellular stalk region and a major truncation of the cytoplasmic domain in a patient with leukocyte adhesion deficiency type 1

Patricia Hixson, C. Wayne Smith, Susan B. Shurin, Michael F. Tosi
Journal: Blood
Blood (2004) 103 (3): 1105–1113.
https://doi.org/10.1182/blood-2003-08-2780
Published: 2004