?-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.
The blood-brain barrier is a huge challenge when it comes to the delivery of therapeutic proteins to treat genetic diseases, injury, and neurodegenerative diseases.
Researchers at Stanford and the Chan Zuckerberg Biohub have developed a methodology to monitor cell expansion and differentiation following targeted genomic modification.