Researchers from the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC) have developed a groundbreaking gene therapy approach that could provide hope for patients with familial hemophagocytic lymphohistiocytosis (FHL), an inherited immune deficiency characterized by severe systemic hyperinflammation.
The study, titled “Precise CRISPR-Cas9 gene repair in autologous memory T cells to treat familial hemophagocytic lymphohistiocytosis,” focuses on repairing mutations in T cells from a mouse model of FHL and two pediatric patients with the condition. The researchers utilized a CRISPR-Cas9 system based on adeno-associated virus (AAV), demonstrating successful repair and paving the way for potential clinical applications in FHL patients and other primary immunodeficiencies with monogenic T cells.
Continue reading… “Breakthrough Gene Therapy Approach Shows Promise for Treating Familial Hemophagocytic Lymphohistiocytosis”