{"id":15768,"date":"2017-06-26T20:50:55","date_gmt":"2017-06-26T23:50:55","guid":{"rendered":"https:\/\/www.infobioquimica.com\/new\/?p=15768"},"modified":"2017-06-26T20:50:55","modified_gmt":"2017-06-26T23:50:55","slug":"scientists-reverse-huntingtons-disease-in-mice-using-crispr","status":"publish","type":"post","link":"https:\/\/www.infobioquimica.com\/new\/2017\/06\/26\/scientists-reverse-huntingtons-disease-in-mice-using-crispr\/","title":{"rendered":"Scientists reverse Huntington’s disease in mice using CRISPR"},"content":{"rendered":"

Huntington\u2019s disease is a fatal neurodegenerative disorder that causes the breakdown of nerve cells in the brain. It\u2019s an inherited condition that typically begins in\u00a0adulthood and is caused by a gene that\u00a0produces proteins that\u00a0are toxic to\u00a0cells in the brain.<\/p>\n

The research, published in the Journal of Clinical Investigation<\/a>, focused on mice engineered to develop Huntington\u2019s disease, with\u00a0symptoms like impaired movement developing when they are\u00a0nine months old. The team then used CRISPR to change the genes of the mice, and within three weeks, the mice were significantly improved, although not back to the level of a healthy mouse.<\/p>\n

The genetic therapy was delivered to the mice’s brain cells using a virus. This approach called the adeno-associated virus, or AAV, has been successful in CRISPR. The viral carrier was injected in the brain striatum of the mice, the region that controls movement.<\/p>\n

\u201cThe findings open up an avenue for treating Huntington’s as well as other inherited neurodegenerative diseases, although more testing of safety and long-term effects is needed,\u201d senior author Professor Xiao-Jiang Li, from Emory University School of Medicine, said in a statement.<\/p>\n

The potential long-term effects are what makes medical researchers tread carefully when it comes to CRISPR. The potential for CRISPR to be a phenomenal weapon in the medical arsenal is undeniable, but there\u2019s still more that we need to understand.<\/p>\n

What if by suppressing a specific gene we know is causing a disease, another gene is affected? Or what if the change leads to other long-term difficulties? The researchers showed that the gene mutations caused by CRISPR in this setup happened only in the Huntington genes and not in off-target genes.<\/p>\n

\u201cThe long-term effects and safety of injecting AAV in the brain to express CRISPR\/Cas9 remain to be rigorously tested before applying this approach to patients,\u201d Li added.<\/p>\n

There are no current plans to use CRISPR to fight neurodegenerative disorders in humans, although human trials to make cells able to fight cancer is currently underway\u00a0in the USA and in China.<\/p>\n

Source:<\/strong> IFLScience<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

Huntington\u2019s disease is a fatal neurodegenerative disorder that causes the breakdown of nerve cells in the brain. It\u2019s an inherited condition that typically begins in\u00a0adulthood and is caused by a gene that\u00a0produces proteins that\u00a0are toxic to\u00a0cells in the brain. The research, published in the Journal of Clinical Investigation, focused on mice engineered to develop Huntington\u2019s […]<\/p>\n","protected":false},"author":2620,"featured_media":15769,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"twitterCardType":"summary_large_image","cardImageID":0,"cardImage":"","cardTitle":"","cardDesc":"","cardImageAlt":"","cardPlayer":"","cardPlayerWidth":0,"cardPlayerHeight":0,"cardPlayerStream":"","cardPlayerCodec":"","footnotes":""},"categories":[655],"tags":[25888,5503,16558,16560,25896,16562,25892,25894,25884,25886,4567,25890],"class_list":["post-15768","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-noticias-ingles","tag-adeno-associated-virus-aav","tag-cancer-en","tag-cas9-en","tag-crispr-en","tag-crispr-cas9-en","tag-crisprcas9-en","tag-emory-university-school-of-medicine","tag-gene-mutations","tag-huntingtons-disease","tag-neurodegenerative-disorders","tag-virus-en","tag-xiao-jiang-li"],"acf":[],"_links":{"self":[{"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/posts\/15768","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/users\/2620"}],"replies":[{"embeddable":true,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/comments?post=15768"}],"version-history":[{"count":1,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/posts\/15768\/revisions"}],"predecessor-version":[{"id":15770,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/posts\/15768\/revisions\/15770"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/media\/15769"}],"wp:attachment":[{"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/media?parent=15768"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/categories?post=15768"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.infobioquimica.com\/new\/wp-json\/wp\/v2\/tags?post=15768"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}