As direct analysis of DNA became possible, we focused our attention on the remarkable diversity in the structure of the α-globin genes, the numerous mutations that produce the diverse clinical ...

Once the alpha globin genes were deleted, the mice experienced decreased oxygen levels comparable to individuals with severe AT. The mice were flooded with RBC that couldn't transport oxygen ...

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Phase 1 study and Investigator Initiated Trial evaluating KL003 for patients with beta thalassemia demonstrated an encouraging efficacy signal ...