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Zebrafish insights into rare disease linked to cancer

Updated: Jun 17

UK researchers have discovered a potential treatment for a rare genetic bone marrow disorder linked to blood cancers.

GATA2 deficiency is a rare disease where patients produce insufficient numbers of immune cells and are more susceptible to infections and the development of blood cancers.

The study at the University of Birmingham and University of Warwick, used gene editing to create zebrafish with GATA2 deficiency, to look at its effect on stem cells - the raw materials of the body from which all other cells with specialised functions come from.

They saw that these animals had fewer specialised immune cells and became less able to repair DNA damage, increasing the risk of developing blood cancers earlier in life.

Dr Rui Monteiro, at Birmingham said : “This work..will enable further investigation on ways to block, slow down or revert the appearance of blood cancers in GATA2 deficiency patients.”

The study was funded by by the British Heart Foundation and Cancer Research UK.


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