🦠 Imuno MD Peľ na terase, génová terapia a výber biologika pri astme

The FDA and EMA approved the first gene therapy for Wiskott-Aldrich syndrome, an X-linked congenital immunodeficiency disorder characterized by microthrombocytopenia, eczema, and primary immunodeficiency. The therapy called etuvatidigene autotemcel is indicated for patients from 6 months of age with WAS gene mutations who lack suitable bone marrow donors. The treatment involves extracting the patient's own hematopoietic stem cells, genetically modifying them using a lentiviral vector, and reinfusing them. In two clinical trials with 27 patients, severe infections decreased by 93 percent between 6 and 18 months post-treatment and bleeding episodes decreased by 60 percent in the first year. Gene therapy represents a significant advancement in treating congenital immunodeficiency.