New genome editing technology targets and kills blood cancer cells | Leukaemia Foundation

New genome editing technology targets and kills blood cancer cells

Providing hope to people living with blood cancer, Melbourne researchers have developed a new technology which directly kills cancer cells by targeting factors essential for their survival and growth.

Using the technology, current and past Leukaemia Foundation National Research Program grant recipients including Dr Brandon Aubrey, Dr Gemma Kelly, Dr Marco Herold, Prof Andreas Strasser and the entire Walter and Eliza Hall Institute team were able to kill human lymphoma cells by locating and deleting an essential gene for cancer cell survival.

Published in the journal Cell Reports, the research provides a ‘proof of concept’ for using the technology as a direct treatment for human disease arising from genetic ‘errors’.

Dr Aubrey, Dr Kelly and Dr Herold adapted the technology, called CRISPR, to specifically mimic and study blood cancers.

Dr Aubrey, who holds a Leukaemia Foundation Clinical PhD Scholarship and is also a haematologist at The Royal Melbourne Hospital, said the team used the CRISPR technology to target and directly manipulate genes in blood cancer cells.

“Using preclinical models, we were able to kill human Burkitt lymphoma cells by deleting MCL-1, a gene that has been shown to keep cancer cells alive,” he said. “Our study showed that the CRISPR technology can directly kill cancer cells by targeting factors that are essential for their survival and growth.”

Dr Herold said the study showed for the first time that it is possible for CRISPR technology to be used in cancer therapy.

“However, CRISPR is a unique approach that could potentially be used for treating any disease that is caused by genetic mutations. The speed at which we are now able to make specific changes in the DNA will also accelerate basic research discoveries in the lab.”

Dr Herold said CRISPR was a very new technology with many advantages over existing tools.

“CRISPR is a rapid, easy and efficient technology with the best results for genome editing.

“In addition to its very exciting potential for disease treatment, we have shown that it has the potential to identify novel mutations in cancer-causing genes and genes that ‘suppress’ cancer development, which will help us to identify how they initiate or accelerate the development of cancer.

“The technology dramatically shortens the time frame for fundamental research, allowing us to speed up the discoveries that could be translated to better diagnostics and treatments for the community.”


The research was supported by the National Health and Medical Research Council, Leukaemia Foundation, Kay Kendall Leukaemia Fund and Victorian Government.

Last updated on March 11th, 2019

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