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New study demonstrates potential of CRISPR for 'correcting' mutated genes in humans

Researchers have demonstrated that "mutations in human embryos" can be "corrected" using gene editing technology.04 Aug 2017

A pioneering experiment tested whether genes linked to hypertrophic cardiomyopathy, a potentially fatal heart condition, could be repaired using CRISPR – clustered regularly interspaced short palindromic repeats.

According to the study, which has now been published by the Nature journal, 42 of 58 embryos were successfully corrected using the technology.

"The efficiency, accuracy and safety of the approach presented suggest that it has potential to be used for the correction of heritable mutations in human embryos by complementing preimplantation genetic diagnosis," the study said. "However, much remains to be considered before clinical applications, including the reproducibility of the technique with other heterozygous mutations."

Earlier this year, trainee patent attorney Asawari Churi of Pinsent Masons, the law firm behind Out-Law.com, explained that there is a major dispute over the patent rights claimed in CRISPR technology. She said the dispute could complicate the way that the technology is licensed in future.

"The battle to control how CRISPR is applied is likely to be highly lucrative to the winner. Its potential commercial application is valued in the billions of dollars," Churi said. "Because CRISPR has many direct commercial applications, research and development (R&D) has not been stifled by the ongoing patent dispute. However, the dispute is a muddying factor."

According to Churi, the dispute has a number of potential outcomes, including the invalidation or narrowing of patent rights claimed by two rival coalitions of universities, or potential differences in rights enjoyed by the groups from one country to another.

A "chequered" patent landscape for CRISPR could, potentially, force the groups to collaborate so as to reach different markets with their technology, Churi said. In addition, the rival coalitions could find that they need to deploy "bespoke licensing strategies in different territories" due to differences in patent law across the world, and they are also likely to have to grapple with ethical issues in how the technology is applied, she said.

Ultimately, CRISPR could be "superseded by alternative genome editing technologies", with some already showing "some promise", Churi said.