Modelling the t(7;12) Infant Leukaemia using Gene Editing Technology

Excellent progress has been made in the cure of childhood leukaemia in the past 20 years, achieving complete remission in more than 90% of acute lymphoid leukaemia sufferers. However, a proportion of very young patients still die of acute myeloid leukaemia. The cancer cells of these children harbour a specific genetic rearrangement between chromosomes 7 and 12, known as t(7;12). This chromosomal abnormality disrupts the function of two genes: ETV6 (normally involved in the production of blood cells), and HLXB9 (involved in the normal development of the embryo). The exact mechanism that leads to this deadly form of leukaemia due to the interaction of these two genes is still unclear. Due to the short life span of these young patients, availability of bone marrow samples is limited impacting on the number and type of studies possible. An ideal study tool would be an in-vitro cellular model that contains the same genetic characteristics of the leukaemia cells found in the patients. Some pioneer studies have shown the potential of using DNA modification methods (gene-editing technologies) to model human malignancies. We propose to use such technologies to generate a cellular model for the t(7;12) leukaemia. This will help us understand the biology of the disease and identify molecular targets leading to possible new treatment.

Grant Award – Kidscan PhD Studentship (2017-2020)
Funding Award – £40,111
Funding Awarded To – Dr Sabrina Tosi
Research Location – Brunel University
Lead Researcher – Dr Sabrina Tosi