{"id":2532,"date":"2015-04-23T00:00:00","date_gmt":"2015-04-23T07:00:00","guid":{"rendered":"https:\/\/vermont.salk.edu\/news-release\/gene-editing-technique-offers-hope-for-hereditary-diseases\/"},"modified":"2015-04-23T00:00:00","modified_gmt":"2015-04-23T07:00:00","slug":"gene-editing-technique-offers-hope-for-hereditary-diseases","status":"publish","type":"disclosure","link":"https:\/\/www.salk.edu\/zh\/news-release\/gene-editing-technique-offers-hope-for-hereditary-diseases\/","title":{"rendered":"Gene-editing technique offers hope for hereditary diseases"},"content":{"rendered":"<p>\nLA JOLLA\u2013For thousands of women around the globe carrying a mitochondrial disease, having a healthy child can be a gamble. This set of diseases affect mitochondria, tiny powerhouses that generate energy in the body\u2019s cells and are passed exclusively from mother to child.\n<\/p>\n<p>\nWomen wishing to prevent their children from inheriting mitochondrial diseases have typically relied on preimplantation genetic diagnosis to pick the healthiest embryos, but that is no guarantee of having a healthy baby.\n<\/p>\n<p><iframe src=\"\/\/www.youtube.com\/embed\/Vks2jDdWyOE\" frameborder=\"0\" allowfullscreen><\/iframe><\/p>\n<p>\nNow, researchers at Salk Institute have developed a simple technique to eliminate mitochondrial mutations from eggs or early embryos, which has the potential to prevent babies from inheriting mitochondrial diseases. Their approach is described in the April 23, 2015 issue of <a href=\"http:\/\/www.cell.com\/cell\/abstract\/S0092-8674(15)00371-2\" target=\"_blank\"><em>Cell<\/em><\/a>.\n<\/p>\n<p>\n\u201cCurrently, there are no treatments for mitochondrial diseases,\u201d says senior author <a href=\"https:\/\/www.salk.edu\/zh\/faculty\/belmonte.html\/\">Juan Carlos Izpisua Belmonte<\/a>, professor in Salk\u2019s <a href=\"https:\/\/www.salk.edu\/zh\/faculty\/gene_expression_laboratory.html\/\">\u57fa\u56e0\u8868\u8fbe\u5b9e\u9a8c\u5ba4<\/a> and holder of the Roger Guillemin Chair. \u201cOur technology may offer new hope for mitochondrial disease carriers wishing to have children without the disease.\u201d\n<\/p>\n<p>\nLiving cells can have hundreds\u2013or even thousands\u2013of mitochondria, each of which contains their own DNA, a small collection of 37 genes that are essential for the organelle\u2019s function. Mutations in these crucial genes can cause a wide range of diseases and can lead to fatality at birth, a life expectancy of only a few years or devastating symptoms for decades.\n<\/p>\n<div class=\"imageCaption\"><img decoding=\"async\" style=\"border-bottom: 1px #006699 solid;\" alt=\"\" src=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2015\/01\/2077-ATP5-ApaLIFLAG-2-100x.jpg\"><\/p>\n<p>Salk Institute researchers used specialized molecules (called nucleases) to eliminate mutated DNA from mitochondria. This image shows the location of nucleases with mitochondria (yellow) in the egg.<\/p>\n<p><a target=\"_blank\" href=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2015\/02\/2077-ATP5-ApaLIFLAG-2-100x.jpg\">Click here<\/a> for a high-resolution image.<\/p>\n<p>\nImage: Courtesy of the Salk Institute for Biological Studies\n<\/p>\n<\/div>\n<p>\n\u201cMost current approaches are trying to develop treatments for patients who are already suffering from these diseases,\u201d says Alejandro Ocampo, a research associate in Izpisua Belmonte\u2019s lab and one of the first authors of the paper. \u201cInstead, we thought of preventing the transmission of these mutations early in development.\u201d\n<\/p>\n<p>\nIzpisua Belmonte and his colleagues turned to two types of molecules: restriction endonucleases and transcription activator-like effector nucleases (TALENs). These nucleases can be engineered to cut specific strands of DNA, functioning as a type of molecular \u201cscissors.\u201d The Salk team designed nucleases to cut only mitochondrial DNA that contained specific, disease-causing mutations in eggs or embryos, leaving healthy mitochondria intact. <\/p>\n<p>\n\u201cWe might not be able to eliminate one hundred percent of the mutated copies of mitochondrial DNA,\u201d says Pradeep Reddy, another research associate in the Izpisua Belmonte lab and first author of the new paper. \u201cBut you don\u2019t need to eliminate all of the mutated copies: just reducing the percentage significantly enough can prevent the disease in the next generation.\u201d\n<\/p>\n<p>\nAs proof of concept, the scientists, using mice containing two types of mitochondrial DNA, selectively prevented the transmission of one of the types to the next generation using specific nucleases in both eggs and one-cell embryos. Baby mice generated by this approach developed normally to adulthood. In addition, this method let the researchers successfully reduce the levels of mutated mitochondrial DNA responsible for two human mitochondrial diseases.\n<\/p>\n<p>\nIn the United Kingdom, lawmakers have recently approved the use of another technology known as mitochondrial replacement to prevent the transmission of mitochondrial diseases. This technology is based on the transfer of the nuclear genome from one patient\u2019s embryo into donor embryos with healthy mitochondria. \u201cThe clinical application of our technique does not require donor eggs,\u201d says Ocampo. \u201cWe are just performing a single injection into the patient\u2019s egg or one-cell embryo, which is technically easier than mitochondrial replacement.\u201d\n<\/p>\n<p>\nIzpisua Belmonte and colleagues are now investigating the possibility of translating this technology to the clinic in human eggs and embryos.\n<\/p>\n<p>\nOther researchers on the study were Keiichiro Suzuki, Jinping Luo, Atsushi Sugawara, Daiji Okamura, Jun Wu, David Lam, Concepcion Rodriguez Esteban, and Ignacio Sancho-Martinez of the Salk Institute; Sandra R. Bacman, Sion L. Williams, and Carlos T. Moraes of the <a href=\"http:\/\/www.miami.edu\/\" target=\"_blank\">University of Miami<\/a>; Yuji Tsunekawa of the <a href=\"http:\/\/www.cdb.riken.jp\/en\/\" target=\"_blank\">RIKEN Center for Developmental Biology<\/a>; Xiong Xiong and Huimin Zhao of the <a href=\"http:\/\/illinois.edu\/\" target=\"_blank\">University of Illinois at Urbana-Champaign<\/a>; Nuria Montserrat of the <a href=\"http:\/\/www.ibecbarcelona.eu\/\" target=\"_blank\">Institute for Bioengineering of Catalonia<\/a>; Guang-Hui Liu of the <a href=\"http:\/\/english.cas.cn\/\" target=\"_blank\">Chinese Academy of Sciences<\/a>; Dolors Manau, Salva Civico, Francesc Cardellach and Josep Maria Campistol of the <a href=\"http:\/\/www.ub.edu\/web\/ub\/en\/\" target=\"_blank\">University of Barcelona<\/a>; and Maria del Mar O\u2019Callaghan and Jaime Campistol of the <a href=\"http:\/\/www.hsjdbcn.org\/\" target=\"_blank\">Hospital Sant Joan de D\u00e9u Barcelona<\/a>.\n<\/p>\n<p>\nThe work was supported by the <a href=\"http:\/\/helmsleytrust.org\/\" target=\"_blank\">Leona M. and Harry B. Helmsley Charitable Trust<\/a>, the <a href=\"http:\/\/www.nih.gov\/\" target=\"_blank\">National Institutes of Health<\/a>, the <a href=\"http:\/\/www.most.gov.cn\/eng\/programmes1\/200610\/t20061009_36223.htm\" target=\"_blank\">National Basic Research Program of China<\/a>, the Strategic Priority Research Program of the Chinese Academy of Sciences, the <a href=\"http:\/\/www.nsfc.gov.cn\/publish\/portal1\/\" target=\"_blank\">National Natural Science Foundation of China<\/a>, the <a href=\"http:\/\/www.thejdmfund.org\/\" target=\"_blank\">JDM Fund<\/a>, the <a href=\"http:\/\/www.mda.org\/\" target=\"_blank\">Muscular Dystrophy Association<\/a>, the <a href=\"http:\/\/www.umdf.org\/site\/c.8qKOJ0MvF7LUG\/b.7929671\/k.BDF0\/Home.htm\" target=\"_blank\">United Mitochondrial Disease Foundation<\/a>, the <a href=\"http:\/\/www.floridahealth.gov\/\" target=\"_blank\">Florida Department of Health<\/a> and the <a href=\"http:\/\/www.mathersfoundation.org\/\">G. Harold and Leila Y. Mathers Charitable Foundation<\/a>.\n<\/p>\n<p><strong>About the Salk Institute for Biological Studies:<\/strong><br \/>\nThe Salk Institute for Biological Studies is one of the world&#8217;s preeminent basic research institutions, where internationally renowned faculty probes fundamental life science questions in a unique, collaborative and creative environment. Focused both on discovery and on mentoring future generations of researchers, Salk scientists make groundbreaking contributions to our understanding of cancer, aging, Alzheimer&#8217;s, diabetes and infectious diseases by studying neuroscience, genetics, cell and plant biology, and related disciplines. <\/p>\n<p>Faculty achievements have been recognized with numerous honors, including Nobel Prizes and memberships in the National Academy of Sciences. Founded in 1960 by polio vaccine pioneer Jonas Salk, MD, the Institute is an independent nonprofit organization and architectural landmark.<\/p>","protected":false},"featured_media":0,"template":"","faculty":[85],"disease-research":[176],"class_list":["post-2532","disclosure","type-disclosure","status-publish","hentry","faculty-juan-carlos-izpisua-belmonte","disease-research-mitochondrial-disease"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene-editing technique offers hope for hereditary diseases - Salk Institute for Biological Studies<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.salk.edu\/zh\/news-release\/gene-editing-technique-offers-hope-for-hereditary-diseases\/\" \/>\n<meta property=\"og:locale\" content=\"zh_CN\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene-editing technique offers hope for hereditary diseases - Salk Institute for Biological Studies\" \/>\n<meta property=\"og:description\" content=\"LA JOLLA\u2013For thousands of women around the globe carrying a mitochondrial disease, having a healthy child can be a gamble. 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