Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Article ID	Journal	Published Year	Pages	File Type
8963699	Journal of Allergy and Clinical Immunology	2018	22 Pages	PDF

Abstract

Our data show that LV-mediated GT for patients with OS significantly ameliorates the immunodeficiency, even in an inflammatory environment.

Keywords

Lin−RAG B cell–activating factor Lineage negative HSCT SCID VCN UCOE TCr PID BAFF Lentiviral vector autoimmune regulator autoimmunity Omenn syndrome bone marrow wild-type AIRE Hematopoietic stem cell transplantation Gene therapy Recombination-Activating Gene primary immunodeficiency severe combined immunodeficiency T-cell receptor

Related Topics

Life Sciences Immunology and Microbiology Immunology

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Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Authors

Valentina PhD, Maria Carmina PhD, Elena PhD, Sara MSc, Marita PhD, Elena MSc, Luigi P. MD, PhD, Lucia MSc, Rosita MSc, Barbara PhD, Monica BSc, Paola PhD, Paolo PhD, Kerry MSc, NicolÃ² BSc, Luigi D. MD, Niek P. PhD, Gerard PhD, Anna MD,