Stanford researchers have developed AZD7648, a novel DNA-PK inhibitor that enhances HDR efficiency in CRISPR-Cas9 gene editing by shifting DNA repair from the error-prone NHEJ pathway to the precise HDR pathway, significantly improving gene targeting outcomes in human cells fo
Stanford researchers have developed a new gene editing approach that enables red blood cell-specific gene expression for the treatment of enzyme deficiencies.
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.
?-thalassemia is a devastating blood disorder caused by mutations in the HBB gene encoding ?-globin, where treatment involves lifelong, costly management of the resulting lack of hemoglobin and hemolytic anemia.
Researchers at Stanford have demonstrated the first method of its kind for treating cystic fibrosis (CF) using regenerated airway stem cells embedded on a biocompatible scaffold.
Researchers at Stanford have developed a potentially curative treatment strategy for alpha-thalassemia, one of the most common autosomal recessive disorders in the world involving the genes HBA1 and/or HBA2.
IPEX syndrome is a severe autoimmune disease with limited treatment options caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation.
Researchers at Stanford have developed gene editing methods for modifying hematopoietic stem and progenitor cells (HSPCs) to express truncated forms of the erythropoietin receptor (tEPOR).
Polycythemia vera is a rare blood cancer characterized by the hyperproliferation of red blood cells, leading to coagulation events like strokes and heart attacks.
Researchers at Stanford and the Chan Zuckerberg Biohub have developed a methodology to monitor cell expansion and differentiation following targeted genomic modification.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an efficacious therapy for patients with life-threatening leukemias, but its use has been hindered by the limited availability of donors with matching HLA. Graft manipulation by removing ??
Researchers at Stanford have developed methods to overcome the limited packaging capacity of adeno-associated virus (AAV) vectors and enable their use in integration of large transgenes.