Article
Gene editing of hematopoietic stem cells restores T-cell response in familial hemophagocytic lymphohistiocytosis.
The Journal of allergy and clinical immunology - 1 Jan 2024
Dettmer-Monaco Viviane, Weißert Kristoffer, Ammann Sandra, Monaco Gianni, Lei Lei, Gräßel Linda, Rhiel Manuel, Rositzka Julia, Kaufmann Masako M, Geiger Kerstin, Andrieux Geoffroy, Lao Jessica, Thoulass Gudrun, Schell Christoph, Boerries Melanie, Illert Anna L, Cornu Tatjana I, Ehl Stephan, Aichele Peter, Cathomen Toni
Abstract excerpt
BACKGROUND: Hemophagocytic lymphohistiocytosis (HLH) is a hyperinflammatory disorder characterized by a life-threatening cytokine storm and immunopathology. Familial HLH type 3 (FHL3) accounts for approximately 30% of all inborn HLH cases worldwide. It is caused by mutations in the UNC13D gene that result in impaired degranulation of cytotoxic vesicles and hence compromised T-cell- and natural...
Topics
- Humans
- Mice
- Animals
- Lymphohistiocytosis, Hemophagocytic
- T-Lymphocytes
- Gene Editing
- Mutation
- Lymphocytic choriomeningitis virus
- Hematopoietic Stem Cells