Gene-editing tool CRISPR found to cause unintentional mutations in live animal models
pharmafile | May 30, 2017 | News story | Manufacturing and Production, Research and Development | crispr
Gene-editing has been heralded as a wonder tool which could lead to huge advances in a wide range of treatment areas, potentially leading to a cure for cancer or even blindness. The technique showing the most promise is CRISPR using Cas9, a protein which acts as a ‘molecular cleaver’, in order to cut DNA and remove, edit or replace genomes. While there has been much discussion and excitement over the clinical possibilities presented by the tool, scientists have now discovered potential risks associated with its use.
Researchers at Columbia University had utilised CRISPR to cure blindness in mice by editing their genomes, but in doing so, it was found that the technique had led to two of the subjects developing over 1,500 unintended mutations and over 100 larger deletions and insertions of genetic material – even one of which could have wide-reaching implications.
These inadvertent effects have in the past been predicted using a computer algorithm and then assessed in isolated DNA sections, and while this was sufficiently effective in simpler tissue samples, it was found that live animal models instead required full genomic sequencing.
None of the mutations observed in the mice were caught by these algorithms, but they did not appear to have negative effects on the animals.
“We feel it’s critical that the scientific community consider the potential hazards of all off-target mutations caused by CRISPR,” explained researcher Professor Stephen Tsang. “Researchers who aren’t using whole genome sequencing to find off-target effects may be missing potentially important mutations.”
He continued: “We hope our findings will encourage others to use whole-genome sequencing as a method to determine all the off-target effects of their CRISPR techniques and study different versions for the safest, most accurate editing.”
Trials of the CRISPR-Cas9 technique are currently being tested in human trials in China, and US-based trials are due to begin later this year. Despite these findings, the researchers maintain that they are still optimistic regarding the technology and are now working to refine its use.
Matt Fellows
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