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LONDON (Reuters) – Scientists studying the effects of the potentially revolutionary CRISPR / Cas9 gene editing tool have discovered that it can cause unexpected genetic damage that can lead to changes dangerous in some cells.
The findings, published in the journal Nature Biotechnology on Monday, have implications for the safety of gene therapies that are being developed using CRISPR / Cas9 – a type of molecular scissor technology that can be used to modify the DNA.
They also add to the findings published last month that suggest that the CRISPR gene editing tool could inadvertently increase the risk of cancer in certain cells.
"We found that changes in DNA have been seriously underestimated before," said Allan Bradley, a professor at the Wellcome Sanger Institute in Britain, who co-directed the research published on Monday.
He urged "whoever thinks to use this technology for gene therapy" to proceed with great caution, and to watch very carefully "to check for possible harmful effects".
CRISPR / Cas9 is one of the most recent genome editing tools. He can edit sections of the DNA in cells by cutting at specific points and introducing changes to that place. It is already widely used in scientific research and is considered by many to be a promising way to create potential genome editing treatments for diseases such as HIV, cancer or sickle cell disease.
Experts say treatments like these could inactivate a gene causing the disease, or correct a genetic mutation, but much more research is still needed to make sure the techniques are safe.
The Bradley team conducted a comprehensive systematic study on human and mouse cells and found that CRISPR / Cas9 frequently induced extensive mutations, including large genetic rearrangements such as deletions and insertions of # 39; DNA.
These could lead to the activation or deactivation of important genes – as predicted by therapies – but could also have major unexpected implications, according to scientists.
They warned that some of the changes observed in this study were too far from the target site to be resumed with standard badysis and test methods. Robin Lovell-Badge, stem cell specialist at the Francis Crick Institute of Britain, said the work has highlighted the need for very careful work when editing the genome to verify that the alterations of the DNA sequence are the same. and only those that were designed to happen. "
" But the results give no reason to panic or lose confidence in the methods when they are conducted by those who know what they are doing ", was he added.
Editing by Richard Balmforth
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