Harnessing RNA interference in gene therapy vectors for β-thalassaemia [ 2018 - ]

Research Grant

Researchers: A/Pr Saovaros Svasti ,  Dr Emmanuel Payen ,  Prof Philippe Leboulch ,  Prof Suthat Fucharoen

Brief description There is an urgent need to develop safe and effective treatments for β-thalassaemia. We anticipate that α-globin-specific RNAi sequences will synergise with β-globin transgene expression to achieve balanced α-/β-globin ratio in a clinical setting. Given that one of the major issues with current gene therapy vectors is achieving high levels of expression, we believe this will be a more effective gene therapy strategy than β-globin transgene expression alone.

Funding Amount $709,259.00

Funding Scheme Project Grants

Notes Standard Project Grant