{"id":11547,"date":"2016-11-16T00:00:26","date_gmt":"2016-11-16T08:00:26","guid":{"rendered":"https:\/\/vermont.salk.edu\/?post_type=disclosure&#038;p=11547"},"modified":"2024-01-30T15:24:21","modified_gmt":"2024-01-30T23:24:21","slug":"new-gene-editing-technology-partially-restores-vision-blind-animals","status":"publish","type":"disclosure","link":"https:\/\/www.salk.edu\/de\/news-release\/new-gene-editing-technology-partially-restores-vision-blind-animals\/","title":{"rendered":"New gene-editing technology partially restores vision in blind animals"},"content":{"rendered":"<p>LA JOLLA\u2014Salk Institute researchers have discovered a holy grail of gene editing\u2014the ability to, for the first time, insert DNA at a target location into the non-dividing cells that make up the majority of adult organs and tissues. The technique, which the team showed was able to partially restore visual responses in blind rodents, will open new avenues for basic research and a variety of treatments, such as for retinal, heart and neurological diseases.<\/p>\n<div class=\"row\" style=\"\"><div class=\"col-md-8 col-md-push-2\"><div class=\"video-anchor\" id=\"video-7-pyDQ1akSM\"><\/div><div class=\"embed-responsive embed-responsive-16by9\"> <iframe class=\"embed-responsive-item\" src=\"\/\/www.youtube.com\/embed\/7-pyDQ1akSM?rel=0\" webkitallowfullscreen mozallowfullscreen allowfullscreen><\/iframe><\/div><!-- .embed-responsive --><\/div><!-- .col-md-*size --><\/div><!-- .\/row -->\n<p>\u201cWe are very excited by the technology we discovered because it\u2019s something that could not be done before,\u201d says <a href=\"https:\/\/www.salk.edu\/de\/scientist\/juan-carlos-izpisua-belmonte\/\">Juan Carlos Izpisua Belmonte<\/a>, a professor in Salk\u2019s Gene Expression Laboratory and senior author of the paper published on November 16, 2016 in <a href=\"http:\/\/www.nature.com\/nature\/journal\/vaop\/ncurrent\/full\/nature20565.html\" target=\"_blank\" rel=\"noopener\"><em>Natur<\/em><\/a>. \u201cFor the first time, we can enter into cells that do not divide and modify the DNA at will. The possible applications of this discovery are vast.\u201d<\/p>\n<p>Until now, techniques that modify DNA\u2014such as the CRISPR-Cas9 system\u2014have been most effective in dividing cells, such as those in skin or the gut, using the cells\u2019 normal copying mechanisms. The new Salk technology is ten times more efficient than other methods at incorporating new DNA into cultures of dividing cells, making it a promising tool for both research and medicine. But, more importantly, the Salk technique represents the first time scientists have managed to insert a new gene into a precise DNA location in adult cells that no longer divide, such as those of the eye, brain, pancreas or heart, offering new possibilities for therapeutic applications in these cells.<\/p>\n<p>To achieve this, the Salk researchers targeted a DNA-repair cellular pathway called NHEJ (for \u201cnon-homologous end-joining\u201d), which repairs routine DNA breaks by rejoining the original strand ends. They paired this process with existing gene-editing technology to successfully place new DNA into a precise location in non-dividing cells.<\/p>\n<figure id=\"attachment_11550\"  class=\"wp-caption alignright\"><img loading=\"lazy\" decoding=\"async\" width=\"458\" height=\"458\" class=\"img-responsive wp-image-11550 size-col-md-5\" src=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-458x458.jpg\" alt=\"gfp-mche-dapi-11stks-10um_016-hr\" srcset=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-458x458.jpg 458w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-150x150.jpg 150w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-300x300.jpg 300w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-768x768.jpg 768w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-1024x1024.jpg 1024w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-767x767.jpg 767w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-147x147.jpg 147w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-585x585.jpg 585w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-553x553.jpg 553w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-750x750.jpg 750w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr-945x945.jpg 945w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr.jpg 2000w\" sizes=\"auto, (max-width: 458px) 100vw, 458px\" \/><figcaption class=\"wp-caption-text\">Pictured is a part of the adult mouse brain. Cell nuclei are blue and genome-edited neurons are green.<\/p>\n<p><a href=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-hr.jpg\" target=\"_blank\" rel=\"noopener\">Klicken Sie hier<\/a> for a high-resolution image<\/p>\n<p>Kredit: Salk Institut<\/figcaption><\/figure>\n<p>\u201cUsing this NHEJ pathway to insert entirely new DNA is revolutionary for editing the genome in live adult organisms,\u201d says Keiichiro Suzuki, a senior research associate in the Izpisua Belmonte lab and one of the paper\u2019s lead authors. \u201cNo one has done this before.\u201d<\/p>\n<p>First, the Salk team worked on optimizing the NHEJ machinery for use with the CRISPR-Cas9 system, which allows DNA to be inserted at very precise locations within the genome. The team created a custom insertion package made up of a nucleic acid cocktail, which they call HITI, or homology-independent targeted integration. Then they used an inert virus to deliver HITI\u2019s package of genetic instructions to neurons derived from human embryonic stem cells.<\/p>\n<p>\u201cThat was the first indication that HITI might work in non-dividing cells,\u201d says Jun Wu, staff scientist and co-lead author. With that feat under their belts, the team then successfully delivered the construct to the brains of adult mice. Finally, to explore the possibility of using HITI for gene-replacement therapy, the team tested the technique on a rat model for retinitis pigmentosa, an inherited retinal degeneration condition that causes blindness in humans. This time, the team used HITI to deliver to the eyes of 3-week-old rats a functional copy of Mertk, one of the genes that is damaged in retinitis pigmentosa. Analysis performed when the rats were 8 weeks old showed that the animals were able to respond to light, and passed several tests indicating healing in their retinal cells.<\/p>\n<p>\u201cWe were able to improve the vision of these blind rats,\u201d says co-lead author Reyna Hernandez-Benitez, a Salk research associate. \u201cThis early success suggests that this technology is very promising.\u201d<\/p>\n<p>The team\u2019s next steps will be to improve the delivery efficiency of the HITI construct. As with all genome editing technologies, getting enough cells to incorporate the new DNA is a challenge. The beauty of HITI technology is that it is adaptable to any targeted genome engineering system, not just CRISPR-Cas9. Thus, as the safety and efficiency of these systems improve, so too will the usefulness of HITI.<\/p>\n<figure id=\"attachment_11552\"  class=\"wp-caption alignleft\"><img loading=\"lazy\" decoding=\"async\" width=\"458\" height=\"335\" class=\"img-responsive wp-image-11552 size-col-md-5\" src=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-458x335.jpg\" alt=\"jun-wu-reyna-hernandez-benitez-keiichiro-suzuki-juan-carlos-izpisua-belmonte0x8c5733\" srcset=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-458x335.jpg 458w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-300x220.jpg 300w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-768x562.jpg 768w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-1024x749.jpg 1024w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-147x108.jpg 147w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-585x428.jpg 585w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-553x405.jpg 553w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-750x549.jpg 750w, https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733-945x692.jpg 945w\" sizes=\"auto, (max-width: 458px) 100vw, 458px\" \/><figcaption class=\"wp-caption-text\">From left: Jun Wu, Reyna Hernandez-Benitez, Keiichiro Suzuki and Juan Carlos Izpisua Belmonte<\/p>\n<p><a href=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/Jun-Wu-Reyna-Hernandez-Benitez-Keiichiro-Suzuki-Juan-Carlos-Izpisua-Belmonte0X8C5733.jpg\">Klicken Sie hier<\/a> f\u00fcr ein hochaufl\u00f6sendes Bild. <\/p>\n<p>Kredit: Salk Institut<\/figcaption><\/figure>\n<p>\u201cWe now have a technology that allows us to modify the DNA of non-dividing cells, to fix broken genes in the brain, heart and liver,\u201d says Izpisua Belmonte. \u201cIt allows us for the first time to be able to dream of curing diseases that we couldn\u2019t before, which is exciting.\u201d<\/p>\n<p>Other researchers on the study were Euiseok J. Kim, Fumiyuki Hatanaka, Mako Yamamoto, Toshikazu Araoka, Masakazu Kurita, Tomoaki Hishida, Mo Li, Emi Aizawa, April Goebl, Rupa Devi Soligalla, Concepcion Rodriguez Esteban, Travis Berggren and Edward M. Callaway of the Salk Institute; Yuji Tsunekawa and Fumio Matsuzaki of <a href=\"http:\/\/www.cdb.riken.jp\/en\/\" target=\"_blank\" rel=\"noopener\">RIKEN Center for Developmental Biology<\/a>; Pierre Magistretti of <a href=\"https:\/\/www.kaust.edu.sa\/en\" target=\"_blank\" rel=\"noopener\">King Abdullah University of Science and Technology<\/a>; Jie Zhu, Tingshuai Jiang, Xin Fu, Maryam Jafari and Kang Zhang of <a href=\"http:\/\/eyesite.ucsd.edu\/\" target=\"_blank\" rel=\"noopener\">Shiley Eye Institute<\/a> und <a href=\"http:\/\/igm.ucsd.edu\/\" target=\"_blank\" rel=\"noopener\">Institute for Genomic Medicine<\/a>, <a href=\"https:\/\/ucsd.edu\/\" target=\"_blank\" rel=\"noopener\">University of California San Diego<\/a>; Zhe Li, Shicheng Guo, Song Chen and Kun Zhang of <a href=\"https:\/\/iem.ucsd.edu\/\" target=\"_blank\" rel=\"noopener\">Institute of Engineering in Medicine, University of California San Diego<\/a>; Jing Qu and Guang-Hui Liu of <a href=\"http:\/\/english.cas.cn\/\" target=\"_blank\" rel=\"noopener\">Chinese Academy of Sciences<\/a>; Jeronimo Lajara, Estrella Nu\u00f1ez and Pedro Guillen of <a href=\"http:\/\/www.ucam.edu\/\" target=\"_blank\" rel=\"noopener\">Universidad Catolica San Antonio de Murcia<\/a>; and Josep M. Campistol of the <a href=\"http:\/\/www.ub.edu\/web\/ub\/en\/\" target=\"_blank\" rel=\"noopener\">University of Barcelona<\/a>.<\/p>\n<p>The work and the researchers involved were supported in part by the <a href=\"https:\/\/www.nih.gov\/\" target=\"_blank\" rel=\"noopener\">Nationale Gesundheitsinstitute<\/a>, <a href=\"http:\/\/www.helmsleytrust.org\/\" target=\"_blank\" rel=\"noopener\">Die Leona M. und Harry B. Helmsley Charitable Trust<\/a>, der <a href=\"http:\/\/www.mathersfoundation.org\/\" target=\"_blank\" rel=\"noopener\">G. Harold and Leila Y. Mathers Charitable Foundation<\/a>, <a href=\"https:\/\/www.mcknight.org\/\" target=\"_blank\" rel=\"noopener\">The McKnight Foundation<\/a>, <a href=\"http:\/\/www.moxiefoundation.org\/\" target=\"_blank\" rel=\"noopener\">The Moxie Foundation<\/a>, der <a href=\"http:\/\/www.fundacionpedroguillen.org\/\" target=\"_blank\" rel=\"noopener\">Dr. Pedro Guillen Foundation<\/a> und <a href=\"http:\/\/www.ucam.edu\/\" target=\"_blank\" rel=\"noopener\">Universidad Catolica San Antonio de Murcia, Spain<\/a>.<\/p>","protected":false},"featured_media":11548,"template":"","faculty":[85],"disease-research":[146,124,335],"class_list":["post-11547","disclosure","type-disclosure","status-publish","has-post-thumbnail","hentry","faculty-juan-carlos-izpisua-belmonte","disease-research-aging-and-regenerative-medicine","disease-research-neuroscience-and-neurological-disorders","disease-research-regeneration"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>New gene-editing technology partially restores vision in blind animals - 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\/de\/news-release\/new-gene-editing-technology-partially-restores-vision-blind-animals\/\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"New gene-editing technology partially restores vision in blind animals - Salk Institute for Biological Studies\" \/>\n<meta property=\"og:description\" content=\"LA JOLLA\u2014Salk Institute researchers have discovered a holy grail of gene editing\u2014the ability to, for the first time, insert DNA at a target location into the non-dividing cells that make up the majority of adult organs and tissues. The technique, which the team showed was able to partially restore visual responses in blind rodents, will open new avenues for basic research and a variety of treatments, such as for retinal, heart and neurological diseases.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.salk.edu\/de\/news-release\/new-gene-editing-technology-partially-restores-vision-blind-animals\/\" \/>\n<meta property=\"og:site_name\" content=\"Salk Institute for Biological Studies\" \/>\n<meta property=\"article:modified_time\" content=\"2024-01-30T23:24:21+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2016\/11\/GFP-mChe-DAPI-11stks-10um_016-767.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"767\" \/>\n\t<meta property=\"og:image:height\" content=\"767\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"5 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/new-gene-editing-technology-partially-restores-vision-blind-animals\\\/\",\"url\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/new-gene-editing-technology-partially-restores-vision-blind-animals\\\/\",\"name\":\"New gene-editing technology partially restores vision in blind animals - 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