251. Intrauterine Haemopoietic Stem Cell Therapy Followed By Postnatal Cell Re-Infusion To Enhance Engraftment in a Murine Thalassaemia Model

Intrauterine haemopoietic stem cell therapy (IUHSCT) can be considered for treating haemoglobinopathies and certain metabolic diseases in early pregnancy, for the ability to easily escalate cell dose for higher engraftment and to target the pre-immune fetus. The potential therapeutic benefits are currently unrealised due to sub-therapeutic cell engraftment from host cell competition for haemopoietic niches or neutralising immune response. We...