{"id":11436,"date":"2017-08-07T08:26:24","date_gmt":"2017-08-07T14:26:24","guid":{"rendered":"https:\/\/www.uchealth.org\/today\/?p=11436"},"modified":"2021-07-08T13:49:51","modified_gmt":"2021-07-08T19:49:51","slug":"gene-editing-opens-new-doors-in-disease-fight","status":"publish","type":"post","link":"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/","title":{"rendered":"Gene editing opens new doors in disease fight"},"content":{"rendered":"<div style=\"margin-top: 0px; margin-bottom: 0px;\" class=\"sharethis-inline-share-buttons\" ><\/div><p>&nbsp;<\/p>\n<p>Mention the word \u201cediting\u201d and most of us think of inserting commas, moving paragraphs around the page, correcting misspellings and the like in reports or manuscripts. But scientific news released last week placed the word far from the realm of red ink and pink erasers.<\/p>\n<p>In a report published Aug. 2 in the journal Nature,\u00a0 researchers from Oregon Health and Science University describe a procedure for editing the DNA of human embryos. The specific target: genetic mutations linked to hypertrophic cardiomyopathy (HCM), which causes heart muscle to thicken and increases the risk of heart failure and death.<\/p>\n<p>The procedure used technology called CRISPR-Cas9, which in very simple terms conducts a surgical strike on errors in an individual\u2019s genetic code. The elements of the attack are twofold. One is a piece of RNA that is designed with online tools to bind to DNA near a specific point \u2013 the mutation \u2013 in the genetic sequence. It\u2019s called \u201cguide RNA.\u201d That\u2019s because it points the second element of the process, the enzyme Cas9, to the mutation. Cas9 cuts the twin strands of the rogue DNA. The cell may then repair itself or a piece of normal, healthy DNA can be inserted.<\/p>\n<p><strong>Get with the program<\/strong><\/p>\n<figure id=\"attachment_11441\" aria-describedby=\"caption-attachment-11441\" style=\"width: 214px\" class=\"wp-caption alignright\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-11441 size-medium\" src=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2017\/08\/07023901\/EXT_080417_Matthew-Taylor.webp\" alt=\"This is a photo of Matthew Taylor, MD, PhD. Taylor is director of the Adult Genetics Clinic at UCHealth University of Colorado Hospital.\" width=\"214\" height=\"300\" srcset=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2017\/08\/07023901\/EXT_080417_Matthew-Taylor.webp 600w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2017\/08\/07023901\/EXT_080417_Matthew-Taylor-214x300.webp 214w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2017\/08\/07023901\/EXT_080417_Matthew-Taylor-107x150.webp 107w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2017\/08\/07023901\/EXT_080417_Matthew-Taylor-200x280.webp 200w\" sizes=\"auto, (max-width: 214px) 100vw, 214px\" \/><figcaption id=\"caption-attachment-11441\" class=\"wp-caption-text\">Matthew Taylor, MD, PhD, director of the Adult Genetics Clinic at UCHealth University of Colorado Hospital, says the new gene editing therapy will help to spur further research at institutions like the Center for Personalized Medicine on the University of Colorado Anschutz Medical Campus. The technology, he adds, is \u201cdisease agnostic\u201d and applicable to addressing a wide variety of genetic illnesses and conditions.<\/figcaption><\/figure>\n<p>The idea that scientists might edit genetic errors that cause human misery is not new, noted <a href=\"https:\/\/www.uchealth.org\/provider\/matthew-taylor-md-phd-internal-medicine\/\">Matthew Taylor, MD, PhD<\/a>, director of the Adult Genetics Lab at UCHealth University of Colorado Hospital and associate director of the Center for Personalized Medicine on the University of Colorado Anschutz Medical Campus. The center is home to a DNA biorepository and a <a href=\"https:\/\/www.uchealth.org\/today\/center-for-personalized-medicine-aims-to-boost-biobank-deposits-spur-genetic-research\/\">budding hub of genetic research aimed at developing targeted therapies for disease<\/a>.<\/p>\n<p>In the relatively recent past, researchers designed proteins that target specific sequences of the genetic code, Taylor said. But CRISPR-Cas9 makes the job easier because of the precision of guide RNA and the relatively short time that it takes to program it.<\/p>\n<p>\u201cThe advantage of CRISPR is that it offers much better specificity for targeting parts of the DNA that we are interested in,\u201d Taylor said. \u201cWe can also design experiments in days and weeks, as opposed to months and months. The technology is a game-changer because we can solve problems with targeting DNA much more efficiently.\u201d<\/p>\n<p><strong>Broad application<\/strong><\/p>\n<p>The significance of the current research lies less in the targeting of HCM specifically than in the proof of the CRISPR-Cas9 editing concept, Taylor said. Genetic tests can already reveal whether embryos from in vitro fertilization have the mutation for HCM. (Embryos from a parent with the HCM mutation have a 50-50 chance of inheriting it.) Those without it are selected for reproduction, thus ensuring that the trait is not passed on to offspring. Gene editing isn\u2019t needed in that instance.<\/p>\n<p>The work of the Oregon researchers, however, validates the broad idea that targeted genetic editing can be done on viable embryos without causing collateral damage.<\/p>\n<p>\u201cThat\u2019s a pretty big deal,\u201d Taylor said, \u201cbut HCM may not be the disease we wind up focusing on.\u201d He noted that biomedical research often yields breakthroughs that are important in treating specific diseases, but have little effect on others. A discovery in muscle disease, for example, generally has no relevance to, say, diabetes, Taylor said.<\/p>\n<p>The CRISPR\/Cas9 approach, by contrast, relies on targeting genetic mutations in general, opening the door to research in many medical areas.<\/p>\n<p>\u201cOnce the technology is applicable to one disease, it will be quickly adaptable to many others,\u201d Taylor said. \u201cIt\u2019s disease-agnostic.\u201d<\/p>\n<p>In addition, Taylor said, the research could give new hope to people with genetic diseases, such as some forms of deafness or blindness, that for some couples could carry a 100 percent of risk of being passed on to children.<\/p>\n<p>\u201cWith these diseases, we don\u2019t have a chance of selecting embryos,\u201d he said. \u201cFor these parents who want biological children, this type of approach could also be applicable.\u201d<\/p>\n<p><strong>Gripping a slippery slope<\/strong><\/p>\n<p>The rapidly expanding world of gene editing continues to raise questions, Taylor acknowledged. For example, the FDA probably will have to decide if each new editing approach will require separate approval, as drugs and other therapies do, or if the agency will approve the general technique for more than a single disease.<\/p>\n<p>The work also raises ethical questions related to what restrictions, if any, should be placed on gene editing. Oregon Health and Science University convened an 11-member Innovative Research and Advisory Panel, which included, among others, clinicians, bioethicists and a lay member. The panel allowed the research that led to the HCM success, but only with significant oversight of \u201cgene correction technologies in human embryos.\u201d<\/p>\n<p>The fears of some that gene editing opens the door to developing \u201cdesigner babies\u201d or master races \u201cseem far away,\u201d Taylor said. But he added that ethical debates over biomedical technology will continue and probably intensify.<\/p>\n<p>\u201cThe writing has been on the wall for a while,\u201d he said. Parents have long been able to get information about the sex of their child and have used it to make reproductive decisions, he noted. \u201cWe\u2019re already doing embryo selection for diseases,\u201d he added.<\/p>\n<p>He predicted that the impact of genetic editing, in various forms, will be incremental, as will be the responses to it.<\/p>\n<p>\u201cThe technology has opened the doors wide to researchers and the development of new therapies,\u201d he said. \u201cIt should be done within the context of biomedical research, with ethical concerns addressed and properly adjudicated.\u201d<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>&nbsp; Mention the word \u201cediting\u201d and most of us think of inserting commas, moving paragraphs around the page, correcting misspellings and the like in reports or manuscripts. But scientific news released last week placed the word far from the realm of red ink and pink erasers. In a report published Aug. 2 in the journal [&hellip;]<\/p>\n","protected":false},"author":2143,"featured_media":18399,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","footnotes":""},"categories":[5],"tags":[9085,138,47,1292,137,263],"class_list":["post-11436","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-innovative-care","tag-biobank","tag-center-for-personalized-medicine","tag-genetic-testing-and-counseling","tag-matthew-taylor","tag-personalized-medicine","tag-university-of-colorado-hospital"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.2 (Yoast SEO v27.2) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Gene editing opens new doors in disease fight - UCHealth Today<\/title>\n<meta name=\"description\" content=\"&nbsp; Mention the word \u201cediting\u201d and most of us think of inserting commas, moving paragraphs around the page, correcting misspellings and the like in reports or manuscripts. But scientific news released last week placed the word far from the realm of red ink and pink erasers...\" \/>\n<meta name=\"robots\" content=\"noindex, follow\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene editing opens new doors in disease fight\" \/>\n<meta property=\"og:description\" content=\"&nbsp; Mention the word \u201cediting\u201d and most of us think of inserting commas, moving paragraphs around the page, correcting misspellings and the like in reports or manuscripts. But scientific news released last week placed the word far from the realm of red ink and pink erasers. In a report published Aug. 2 in the journal [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\" \/>\n<meta property=\"og:site_name\" content=\"UCHealth Today\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/uchealthorg\/\" \/>\n<meta property=\"article:published_time\" content=\"2017-08-07T14:26:24+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2021-07-08T19:49:51+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/09\/07122626\/Fall-colors-Poudre-Canyon-Gateway-natural-area.jpg\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Tyler Smith\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@uchealth\" \/>\n<meta name=\"twitter:site\" content=\"@uchealth\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Tyler Smith\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"5 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\"},\"author\":{\"name\":\"Tyler Smith\",\"@id\":\"https:\/\/www.uchealth.org\/today\/#\/schema\/person\/98c85c0e40c4933eedcec2cd054f349d\"},\"headline\":\"Gene editing opens new doors in disease fight\",\"datePublished\":\"2017-08-07T14:26:24+00:00\",\"dateModified\":\"2021-07-08T19:49:51+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\"},\"wordCount\":986,\"publisher\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/#organization\"},\"image\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/09\/07122626\/Fall-colors-Poudre-Canyon-Gateway-natural-area.webp\",\"keywords\":[\"Biobank\",\"Center for Personalized Medicine\",\"Genetic testing\",\"Matthew Taylor\",\"Personalized medicine\",\"University of Colorado Hospital\"],\"articleSection\":[\"Innovative care\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\",\"url\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\",\"name\":\"Gene editing opens new doors in disease fight - UCHealth Today\",\"isPartOf\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#primaryimage\"},\"image\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/09\/07122626\/Fall-colors-Poudre-Canyon-Gateway-natural-area.webp\",\"datePublished\":\"2017-08-07T14:26:24+00:00\",\"dateModified\":\"2021-07-08T19:49:51+00:00\",\"breadcrumb\":{\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\/\/www.uchealth.org\/today\/gene-editing-opens-new-doors-in-disease-fight\/#primaryimage\",\"url\":\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/09\/07122626\/Fall-colors-Poudre-Canyon-Gateway-natural-area.webp\",\"contentUrl\":\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/09\/07122626\/Fall-colors-Poudre-Canyon-Gateway-natural-area.webp\",\"width\":1200,\"height\":900,\"caption\":\"About 4 miles up the Poudre Canyon is Gateway Natural Area owned by the City of Fort Collins. The park has picnic and play areas and is a great place to take a walk for fall colors. The wide maintained trail leads you along the river to Seaman Reservoir about a mile back. It's a great walk for kids (even on their bike) and strollers. There is a steep 100 yard incline up to the reservoir that may be difficult for some. There are also trails off the main trail leading to overlooks. 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He served as a writer and editor for the Marketing and Communications team at University of Colorado Hospital and UCHealth from 2007 to 2017. More recently, he has reported for and contributed stories to the University of Colorado School of Medicine, the Colorado School of Public Health and the Colorado Bioscience Association.\",\"url\":\"https:\/\/www.uchealth.org\/today\/author\/smiths3\/\"}]}<\/script>\n<!-- \/ Yoast SEO Premium plugin. -->","yoast_head_json":{"title":"Gene editing opens new doors in disease fight - UCHealth Today","description":"&nbsp; Mention the word \u201cediting\u201d and most of us think of inserting commas, moving paragraphs around the page, correcting misspellings and the like in reports or manuscripts. 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The park has picnic and play areas and is a great place to take a walk for fall colors. The wide maintained trail leads you along the river to Seaman Reservoir about a mile back. It's a great walk for kids (even on their bike) and strollers. There is a steep 100 yard incline up to the reservoir that may be difficult for some. There are also trails off the main trail leading to overlooks. 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