Gene editing figures to be part of the future of medicine, but a popular system for it has some room for improvement, researchers say.<\/p>\n
That\u2019s why a team from UC San Diego in La Jolla set out with Yale University researchers to develop a new system for gene editing that they believe may be safer and more efficient than the common current method known as CRISPR.<\/p>\n
CRISPR, short for \u201cclustered regularly interspaced short palindromic repeats,\u201d uses RNA and bacterial proteins to edit DNA. According to UCSD, it was adapted from a method used by bacteria as an immune defense against the DNA of viruses.<\/p>\n
But when scientists use the method to edit human DNA, there can be unintended consequences such as genomic damage, said Aaron Smargon, first author of the study and an assistant project scientist in professor Gene Yeo\u2019s lab at UCSD.<\/p>\n
Several years ago, Smargon and Yeo got to work on a possible alternative that targeted small nuclear RNAs \u2014 RNA molecules that don\u2019t make proteins but are located in the nucleus of cells \u2014 to swap out certain \u201cletters\u201d in the genetic code. Unlike CRISPR, this approach makes more specific, temporary modifications, researchers say.<\/p>\n
The result is a tool that could have \u201cwide applicability to a lot of complex genes that can accumulate a lot of mutations in the general population \u2026 that could be problematic,\u201d Smargon said. \u201cBeyond that, the small nuclear RNA we tested didn\u2019t have off-target effects relative to state-of-the-art [technology], which suggests it could be a safer technology.\u201d<\/p>\n
He said the new tool also is capable of editing pre-mRNA, the precursor to the RNA that encodes protein genes, and long non-coding RNA, which has been \u201cimplicated in a number of diseases including neurodegenerative and cardiovascular,\u201d he said. \u201cSo there are a lot of exciting applications\u201d that could be personalized to treat individual diseases.<\/p>\n
\u201cWe talk about precision medicine, and this is another arrow in the quiver that will, in some ways, make the treatments more effective and safer,\u201d Smargon said. \u201cIn the scientific community, we are focused on developing therapeutics that are safer \u2026 that could really benefit patients the most.\u201d<\/p>\n
Though gene editing and CRISPR have been around for decades, it has been \u201camazing\u201d to see how far the science has come, Smargon\u00a0said.<\/p>\n
\u201cThe CRISPR revolution has been and continues to be exhilarating,\u201d he said. \u201cOur work is adjacent to it because if CRISPR hadn\u2019t happened, our work wouldn\u2019t have happened. It\u2019s been incredible to be part of the community and rapidly learn so much about genetics and learn from really great scientists and mentor new scientists. \u2026 We are seeing the first fruits of this labor.<\/p>\n
\u201cThe benefits of CRISPR research will continue to compound. This century, you are going to see a lot of amazing new discoveries and technologies that will benefit society. The future is definitely bright.\u201d \u2666<\/p>\n","protected":false},"excerpt":{"rendered":"Gene editing figures to be part of the future of medicine, but a popular system for it has…\n","protected":false},"author":2,"featured_media":448934,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[557,267,151094,151092,151093,151095,6711,50053,70,16,15],"class_list":{"0":"post-448933","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-california","9":"tag-genetics","10":"tag-la-jolla","11":"tag-la-jolla-light","12":"tag-la-jolla-light-news","13":"tag-la-jolla-shores","14":"tag-san-diego","15":"tag-san-diego-county","16":"tag-science","17":"tag-uk","18":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/115261593697344089","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/448933","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=448933"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/448933\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/448934"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=448933"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=448933"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=448933"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}