Adelaide DNA editing experiments bring promise of cures for epilepsy and muscular dystrophy
ADELAIDE scientists are experimenting with DNA technology that promises to cure genetic diseases such as epilepsy and muscular dystrophy.
ADELAIDE scientists have been experimenting with DNA technology that promises to cure traumatic genetic diseases such as muscular dystrophy and epilepsy.
The gene editing technique known as CRISPR works like a tiny pair of scissors directed to cut DNA at a precise location.
The faulty DNA can then be removed and repaired, or replaced, restoring its function.
University of Adelaide Professor Paul Thomas is director of the SA Genome Editing Facility at SAHMRI, working on models of human disease.
One example is Duchenne muscular dystrophy, “a devastating condition that puts boys in a wheelchair by age 10 or 12 and the survival rate is low”.
“We’re using CRISPR to cut the diseased gene in the (mouse) muscles and then restore the function of the gene,” Professor Thomas said.
“So even if it doesn’t have perfect function, it has enough to improve the condition considerably, by making enough functional protein.
“I think that would be a wonderful benefit, if we could use CRISPR as a gene therapy tool.”
He said the technique's ultimate success relied on predictable and efficient DNA repair.
“You definitely want to be cutting in the right place, not the wrong place.
Making a genetic change that’s predictable and going to be of benefit to the patient,” Prof Thomas said.
Earlier this year, Professor Thomas challenged North American research on human embryos, providing his own evidence that large sections of DNA had gone missing, with unknown consequences.
“We need to be extremely cautious about ever using this CRISPR-based editing approach in correction of human embryos because we don’t really know what happens a lot of the time after CRISPR makes a break in the chromosome,” he said.
“These unexpected findings mean it may look correct but in reality it’s not correct. It would have disastrous consequences.”
The first CRISPR editing publication in 2012 triggered an explosion of international research and more than 9000 papers with applications in medicine and agriculture.
But the new technique poses problems for regulators, because it doesn’t fit neatly into existing legislation.
Adelaide, state and territory ministers on the Legislative and Governance Forum on Gene Technology will meet today and consider changes.