Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency

Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency

Autosomal recessive (AR) complete interferon-γ receptor 1 (IFN-γR1) deficiency, also known as one genetic etiology of Mendelian susceptibility to mycobacterial disease (MSMD), is a life-threatening congenital disease leading to premature death. Affected patients present a pathognomonic predispositio...

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Bibliographic Details
Main Authors: Katharina Hahn, Liart Pollmann, Juliette Nowak, Ariane Hai Ha Nguyen, Kathrin Haake, Anna-Lena Neehus, Syed F. Hassnain Waqas, Frank Pessler, Ulrich Baumann, Miriam Hetzel, Jean-Laurent Casanova, Ansgar Schulz, Jacinta Bustamante, Mania Ackermann, Nico Lachmann
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
MSMD
IFN-γR1 deficiency
Lentiviral Vectors
Gene Therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300620

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