What is this research looking at?
Chronic myelomonocytic leukaemia (CMML) is a common myeloid malignancy caused by mutations in bone marrow cell DNA (genetic mistakes). CMML patients typically survive only 2 to 3 years from diagnosis and there is an urgent need for new more effective treatments for this disorder.
CMML originates from leukaemia stem cells and only a small number of these cells can be obtained from the bone marrow samples of CMML patients for study. Induced pluripotent stem cell (iPSC) is an exciting technology that allows for the generation of an unlimited supply of patient leukaemic cells for study. iPSC essentially reprograms cells, allowing us to turn any cell of the body into a pluripotent (immature) stem cell.
In our research, we are also using CRISPR/Cas9, a genome editing system. Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change sections of DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Gene mutations can be introduced or corrected in cells using this approach.
Using the combined power iPSC technology and CRISPR/Cas9 genome editing, we have generated new cell-based models of CMML. These cell models will now be used for experiments designed to better understand the molecular mechanisms underlying CMML and for drug screening to identify new drugs as therapies for this disorder.
What could this mean for people with leukaemia?
Our research will provide unique opportunities to provide a better understanding of the biology and progression of CMML and for the identification of new drugs for therapy and potentially cure.
Leuka funding provides crucial financial support that allows us to undertake important translational research for the study of CMMLProfessor Jaqueline Boultwood, University Oxford
Official title of project: Induced pluripotent stem cell/CRISPR-Cas9-based modelling of chronic myelomonocytic leukaemia: molecular pathogenesis, disease progression/prognosis and drug screening