Precision gene editing for the treatment of Fanconi Anaemia. 

2021-2024 (Grant-in-Aid): Precision gene editing for the treatment of Fanconi Anaemia. Dr Lorna McLeman, St Vincent’s Institute of Medical Research.

Fanconi Anaemia is the most common cause of inherited bone marrow failure with a median onset of only eight years of age. Bone marrow failure is fatal in Fanconi Anaemia except for one life saving therapy: a bone marrow transplant.  However, transplants have risks to children, including life threatening toxicities and increased risk of developing secondary cancers. Such risks would be substantially reduced by using gene therapy, which corrects the genetic mutation that causes the bone marrow failure. Recent published clinical trials have proven gene therapy as a potential curative treatment for bone marrow failure in these patients. This project will investigate a new gene editing technology (Prime Editing), with the aim of efficiently correcting Fanconi mutations. If successful, this would provide a new alternative therapeutic option for preventing bone marrow failure in this patient group.