{"id":273832,"date":"2025-11-20T16:04:13","date_gmt":"2025-11-20T16:04:13","guid":{"rendered":"https:\/\/www.newsbeep.com\/uk\/273832\/"},"modified":"2025-11-20T16:04:13","modified_gmt":"2025-11-20T16:04:13","slug":"versatile-gene-editing-tool-could-tackle-a-host-of-diseases","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/uk\/273832\/","title":{"rendered":"versatile gene-editing tool could tackle a host of diseases"},"content":{"rendered":"

\"Artist's<\/p>\n

A cell\u2019s protein-making machinery (artist\u2019s illustration) can be instructed to ignore mutations that would otherwise result in shortened proteins. Credit: Christoph Burgstedt\/Science Photo Library<\/p>\n

A single multipurpose gene-editing tool can correct several genetic conditions by restoring proteins that have been truncated by disease-causing mutations. The method might one day overcome a key stumbling block faced by gene-editing therapies: the need to design a bespoke treatment<\/a> for each disease.<\/p>\n

The new approach, called PERT, combines gene editing<\/a> with engineered RNA molecules that allow protein synthesis to continue even when a mutation in the DNA tells it to stop prematurely. Such mutations are called nonsense mutations, and they comprise nearly one-quarter of known disease-causing DNA variants.<\/p>\n

\"\"<\/p>\n

Powerful CRISPR system inserts whole gene into human DNA<\/p>\n

<\/a><\/p>\n

So far, PERT has overcome disease-linked nonsense mutations in mice and in human cells grown in culture, but further testing and refinement are needed before the approach can be studied in people, says David Liu, a chemical biologist at the Broad Institute in Cambridge, Massachusetts, and a co-author of a paper about the technique.<\/p>\n

If it does prove effective in humans, PERT could drive down the cost and speed up the development of gene-editing therapies<\/a> for many conditions. \u201cDisease-agnostic approaches like this raise a possibility that, I think, would be incredibly exciting for patients,\u201d says Liu. He and his colleagues published their results on 19 November in Nature1<\/a>.<\/p>\n

PERT proposal<\/p>\n

Liu first dreamt up the idea for PERT a few years ago while preparing for an event called science karaoke at his laboratory\u2019s annual retreat. During this event, each member presents an out-of-the-box idea for a project that could send the lab in a new direction. Singing is not required.<\/p>\n

For his project, Liu suggested that the team should investigate synthetic RNA molecules called suppressor tRNAs, which allow a cell\u2019s protein-synthesis machinery to read through a nonsense mutation in the DNA and produce a full-length protein. Suppressor tRNAs, Liu proposed, could be inserted into a genome using a technique called prime editing<\/a>. At the end of his ten-minute presentation, Liu asked his lab if anyone would be interested in taking on the project; a few members volunteered.<\/p>\n

\"\"<\/p>\n

Personalized gene editing helped one baby: can it be rolled out widely?<\/p>\n

<\/a><\/p>\n

As the team worked to develop PERT, other researchers published promising results using suppressor tRNAs without gene editing. One group used a virus to shuttle the tRNA into cells2<\/a>; another encased their tRNAs in fatty particles similar to those used in COVID-19 mRNA vaccines3<\/a>.<\/p>\n

Each of these approaches showed promise in animal models with genetic diseases. But viruses can provoke dangerous immune responses, and fatty particles required repeated doses, says Zoya Ignatova, a biochemist at the University of Hamburg in Germany.<\/p>\n

Gene editing, by contrast, would allow researchers to insert a gene encoding the suppressor tRNA into a recipient\u2019s genome. In theory, no further doses would be needed.<\/p>\n","protected":false},"excerpt":{"rendered":"A cell\u2019s protein-making machinery (artist\u2019s illustration) can be instructed to ignore mutations that would otherwise result in shortened…\n","protected":false},"author":2,"featured_media":273833,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[10],"tags":[55593,59,916,102,4230,10394,4231,90,56,54,55],"class_list":["post-273832","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health","tag-crispr-cas9-genome-editing","tag-gb","tag-genetics","tag-health","tag-humanities-and-social-sciences","tag-molecular-biology","tag-multidisciplinary","tag-science","tag-uk","tag-united-kingdom","tag-unitedkingdom"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts\/273832","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/comments?post=273832"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts\/273832\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/media\/273833"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/media?parent=273832"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/categories?post=273832"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/tags?post=273832"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}