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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Detalles Bibliográficos
Autores principales: Hahn, Katharina, Pollmann, Liart, Nowak, Juliette, Nguyen, Ariane Hai Ha, Haake, Kathrin, Neehus, Anna-Lena, Waqas, Syed F. Hassnain, Pessler, Frank, Baumann, Ulrich, Hetzel, Miriam, Casanova, Jean-Laurent, Schulz, Ansgar, Bustamante, Jacinta, Ackermann, Mania, Lachmann, Nico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184269/
https://www.ncbi.nlm.nih.gov/pubmed/32355867
http://dx.doi.org/10.1016/j.omtm.2020.04.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184269/
https://www.ncbi.nlm.nih.gov/pubmed/32355867
http://dx.doi.org/10.1016/j.omtm.2020.04.002

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