Genome editing of human hematopoietic stem and progenitor cells (HSPCs) has the potential to create a new class of medication for the treatment of inherited and acquired genetic diseases of the blood and immune system.
Introduction: Blood cell transfusion plays a vital role in modern medicineāsupporting surgery, obstetrics, trauma care, and cancer chemotherapy. In the US alone, more than 12 million red-cell units are consumed annually.
Stanford inventors have identified a therapeutic CRISPR-based gene editing strategy that restores expression of Forkhead Box Protien 3 (FOXP3) isoforms that are disrupted in patients with Immunedysregulation, Polyendocrinopathy, Enteropathy X-linked Syndrome (IPE
Polycythemia vera is a rare blood cancer characterized by the hyperproliferation of red blood cells, leading to coagulation events like strokes and heart attacks.