Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome
Articolo
Data di Pubblicazione:
2019
Abstract:
Masiuk et al. use a lentiviral vector designed from endogenous FOXP3 regulatory elements in HSCs to achieve lineage-specific FoxP3 expression. This strategy restores functional Treg cell development from FoxP3 mut HSCs and reverses autoimmunity in vivo. These findings suggest preclinical efficacy for lentiviral gene therapy for the treatment of IPEX syndrome.
Tipologia CRIS:
1.1 Articolo in rivista
Keywords:
autoimmunity; FoxP3; gene therapy; hematopoietic stem cells; IPEX; Tregs; Animals; Diabetes Mellitus, Type 1; Diarrhea; Disease Models, Animal; Forkhead Transcription Factors; Genes, Reporter; Genetic Diseases, X-Linked; Hematopoietic Stem Cells; Humans; Immune System Diseases; Lentivirus; Mice; T-Lymphocytes, Regulatory; Autoimmunity; Cell Lineage; Genetic Therapy
Elenco autori:
Masiuk, K. E.; Laborada, J.; Roncarolo, M. G.; Hollis, R. P.; Kohn, D. B.
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