blood disorder

By unraveling the molecular underpinnings of inherited blood disorders, the Boston Children’s Hospital researcher has provided the basis for therapies now being tested for beta-thalassemia and sickle cell disease.

The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.

Researchers use base-editing to swap out an erroneous nucleotide responsible for a potentially life-threatening blood disorder.

Once implanted in mice, the edited stem cells produced normal hemoglobin.

Scientists use the genome-editing technique to fix a disease-causing mutation in human cell lines.