In utero Gene Therapy (IUGT) for Congenital Blood Disorders
Beta thalassaemia is a genetic blood disease that causes life-threatening anemia. Hematopoietic stem cell (HSC) transplantation successfully cures the disease but in only 30% of patients.
In humans, successes with in utero transplantation using allogeneic haematopoietic stem cells, has been limited to fetuses with severe immunologic defects where there is an effective lack of immune response to allogeneic cells.
We hypothesized that IUGT to the fetal HSC compartment (Liver) using a vector carrying the corrected beta globin gene (GLOBE, Dr Mike Antoniou Kings College London) might cure the disease before birth.
Ideally, having a diagnosis using Non Invasive Prenatal Diagnosis (NIPD) at 10 weeks of gestation will give enough time to administer IUGT and correct the disease before birth.