Fetal stem cell gene therapy

Overview: Inherited genetic disorders are important causes of morbidity and mortality. Prenatal diagnosis is now possible early in pregnancy using the maternal blood. Postnatal stem cell transplantation is limited by the immune response to the cells. Stem cells in the amniotic fluid (AF) cells may be a good cell source for prenatal therapy. Some of these AF stem cells have hematopoietic activity meaning they may be used to treat inherited blood disorders such as thalassaemia or sickle cell anaemia. We are studying how to use AF stem cells to correct inherited anaemias before birth.

Design:

Team: Durrgah L Ramachandra, Stavros P Loukogeorgakis, Dr Panicos Shangaris, Eleni Antoniadou, Sindhu Subramaniam.

Funding: Wellcome Trust, Sparks, Rosetrees Trust

Links: Wellcome Trust, Sparks, Rosetrees Trust, UK Thalassaemia Society