CRISPR has revolutionised DNA modification, changing how we think about treating disease. But just what is this new technology and how is it being applied at the Institute?

CRISPR is an acronym for ‘clustered regular interspaced short palindromic repeats’. It is a DNA process, first discovered in bacteria, that is a crucial component of the immune systems of these simple life forms.

It evolved in single-celled microbes as a way of fighting viruses: when viruses attack a cell, they inject their own DNA into the cell. The cell responds with CRISPR, which makes a cut in the DNA, disabling the attack of the virus.

Australian expertise

In recent years scientists have learned how to harness CRISPR technology in the lab to make precise changes in the genes of organisms as diverse as fruit flies, fish, mice, plants and human cells.

Thanks to support from the Australian Cancer Research Foundation, the Institute has one of the most advanced CRISPR laboratories in Australia, established and led by Dr Marco Herold.

Institute scientists have adapted the CRISPR gene-editing technology to directly target any genes in cells derived from blood cancers for the validation of potential new drug targets.

Research published in 2015 by Dr Herold and colleagues Dr Brandon Aubrey, Dr Gemma Kelly and Dr Andrew Kueh showed that the CRISPR technology can be used to kill cancer cells in the laboratory by targeting factors that are essential for cancer cell survival and growth.

Dr Herold said pharmaceutical companies around the world were already investing millions of dollars to develop CRISPR as a tool for treating genetic diseases such as cancer.

Treating genetic disease

“There is a lot of excitement and a significant number of resources being invested worldwide to use CRISPR technology for treating patients. It is possible for CRISPR technology to be used in cancer therapy,” Dr Herold said.

“It is possible for CRISPR technology to be used in cancer therapy.”

“In our study, we showed for the first time in pre-clinical models of cancer that it may be possible for CRISPR technology to be used in cancer therapy. CRISPR is a unique approach that has the potential for use in treating disease that is caused by a single genetic mutation.

“The speed at which we are now able to make specific changes in the DNA will also accelerate basic research discoveries in the lab.”

Using the technology, the researchers were able to kill human-derived lymphoma cell lines in the laboratory by locating and deleting an essential gene for cancer cell survival.

The research, published in the journal Cell Reports, provided ‘proof of concept’ for using the technology as a treatment for human diseases arising from genetic ‘errors’.

“In addition to its very exciting potential for disease treatment, we have shown in cells derived from blood cancers 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,” Dr Herold said.

“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.”

More than 100 research groups from Australia and around the world have sought Dr Herold’s expertise and are working with the laboratory to adapt the technology for their own research. 

Dr Herold is the Broomhead Centenary Fellow in genome engineering.