New study gives a note of caution in the development of CRISPR gene therapies



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The discovery of gene editing technology CRISPR has led many people to believe that we would soon be able to modify our DNA with very high accuracy. But a new study has shown that it can cause more unintended damage to the genome than thought, which casts doubt on its potential use in humans.

Since the researchers demonstrated at the beginning of this decade that the immune system technology of bacteria, could be used to make precise modifications to the DNA of animals and humans, scientists and technology companies have dreamed of potential applications.

This kind of fine control on the genome could be used in medicine to treat the genetic component of many diseases; to deceive the algae by producing biofuel for us; strengthen efforts to genetically modify crops and livestock for better yields; and even potentially adjust our own genomes to increase our capabilities.

The technology uses an enzyme to cut the DNA of a cell in a specific place and then relies on the cell to glue the DNA together again. This can either be used to hit specific genes or even introduce new genetic materials at the cutting site.

But in an article published in Nature Biotechnology on Monday, researchers at Wellcome The Sanger Institute in the UK described how CRISPR publishes two types of mouse cells and a type Human cell can often cause large deletions or rearrangements of DNA around the target site.

This is not necessarily a problem for many uses of CRISPR. As for editing the genomes of microorganisms or plants, humans worry that this type of damage to the DNA could cause cancer.

Part of the reason why this question went unnoticed I looked for the wrong kind of mutations. They tested small mutations near the target, so that larger ones or those that are farther away do not appear in standard tests.

The publication of the research saw shares of genetic engineering companies fall last Monday. Most wanted to minimize the importance of the results, suggesting that their methods were significantly different from those used by the study authors, that they used different types of cells that would not be affected by the problem or simply that they were looking for similar effects

But the study adds to other recent news suggesting that the technology might not be quite ready for prime time. A study last month showed that human embryonic stem cells successfully edited with CRISPR were more likely to have mutations in a gene that helps prevent cancer. And early in the year, a Stanford document on the biorXiv pre-print server – a research repository that has not yet been peer reviewed – suggested that the technology might not work. on many humans because we already have CRISPR bacteria is derived from. (Although there are some noticeable warnings about this and what it would mean for the future of CRISPR therapies is not yet clear.)

However, it is unlikely that it slows down the pace of field research. China has been using technology on human patients since 2015, and the first US and European clinical trials are planned for this year. It is also unlikely that the problem affects newer and more advanced CRISPR techniques that alter simple DNA letters rather than altering entire pieces of code or turning on or off individual genes.

But another question is whether results based therapies. A cancer risk, as small as it may be, could easily move patients away from the approach, but the potential for change in these therapies could also convince them that the risk is worth it.

For their part, the researchers say that the study should act as a warning for those considering using CRISPR in humans to be more vigilant about potential harms.

"We found that changes in DNA were seriously underestimated before," Professor Allan Bradley of the Wellcome Sanger Institute said in a press release. "It is important that anyone who is considering using this technology for gene therapy proceeds with caution, and examines very carefully the possible harmful effects."

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