{"id":1396,"date":"2019-12-20T15:42:34","date_gmt":"2019-12-20T15:42:34","guid":{"rendered":"https:\/\/blogs.bmj.com\/jmg\/?p=1396"},"modified":"2026-02-24T00:20:41","modified_gmt":"2026-02-24T00:20:41","slug":"gene-editing-prospects-for-treating-inherited-retinal-diseases","status":"publish","type":"post","link":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/","title":{"rendered":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia)"},"content":{"rendered":"<p>Retinal diseases can result from genetic mutations or combined risk factors, all causing vision impairment progressing to blindness. To date, available therapeutic approaches result in inefficient and expensive treatments. The CRISPR-Cas9 system, an RNA-guided genome editing technology, may be applied to precisely correct or remove disease-related genetic mutations, or induce permanent inhibition of factors promoting retinal degeneration. In this review, we will describe the CRISPR\/Cas mechanism, as well as delivery tools. Also, we will focus on applications of CRISPR\/Cas genome editing in the retina and in patient-derived induced pluripotent stem cells, highlighting the potential and future challenges of this technology to tackle retinal diseases. (<a href=\"https:\/\/jmg.bmj.com\/content\/early\/2019\/12\/18\/jmedgenet-2019-106473\">https:\/\/jmg.bmj.com\/content\/early\/2019\/12\/18\/jmedgenet-2019-106473<\/a> )<\/p>\n<p>&nbsp;<!--TrendMD v2.4.8--><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Retinal diseases can result from genetic mutations or combined risk factors, all causing vision impairment progressing to blindness. To date, available therapeutic approaches result in inefficient and expensive treatments. The CRISPR-Cas9 system, an RNA-guided genome editing technology, may be applied to precisely correct or remove disease-related genetic mutations, or induce permanent inhibition of factors promoting [&#8230;]<\/p>\n<p><a class=\"btn btn-secondary understrap-read-more-link\" href=\"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/\">Read More&#8230;<\/a><\/p>\n","protected":false},"author":123,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-1396","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog<\/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:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog\" \/>\n<meta property=\"og:description\" content=\"Retinal diseases can result from genetic mutations or combined risk factors, all causing vision impairment progressing to blindness. To date, available therapeutic approaches result in inefficient and expensive treatments. The CRISPR-Cas9 system, an RNA-guided genome editing technology, may be applied to precisely correct or remove disease-related genetic mutations, or induce permanent inhibition of factors promoting [...]Read More...\" \/>\n<meta property=\"og:url\" content=\"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/\" \/>\n<meta property=\"og:site_name\" content=\"JMG Contact blog\" \/>\n<meta property=\"article:published_time\" content=\"2019-12-20T15:42:34+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-02-24T00:20:41+00:00\" \/>\n<meta name=\"author\" content=\"hqqu\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@HuiQiQu\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"hqqu\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/\"},\"author\":{\"name\":\"hqqu\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/person\\\/be0250f8d5b52412c3e7c222dabd591b\"},\"headline\":\"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia)\",\"datePublished\":\"2019-12-20T15:42:34+00:00\",\"dateModified\":\"2026-02-24T00:20:41+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/\"},\"wordCount\":128,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#organization\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/\",\"name\":\"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#website\"},\"datePublished\":\"2019-12-20T15:42:34+00:00\",\"dateModified\":\"2026-02-24T00:20:41+00:00\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2019\\\/12\\\/20\\\/gene-editing-prospects-for-treating-inherited-retinal-diseases\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia)\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#website\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/\",\"name\":\"JMG Contact blog\",\"description\":\"JMG Contact blog\",\"publisher\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#organization\",\"name\":\"JMG Contact blog\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/files\\\/2017\\\/10\\\/blog-logo-jmg.png\",\"contentUrl\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/files\\\/2017\\\/10\\\/blog-logo-jmg.png\",\"width\":300,\"height\":34,\"caption\":\"JMG Contact blog\"},\"image\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/logo\\\/image\\\/\"}},{\"@type\":\"Person\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/person\\\/be0250f8d5b52412c3e7c222dabd591b\",\"name\":\"hqqu\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g\",\"url\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g\",\"contentUrl\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g\",\"caption\":\"hqqu\"},\"description\":\"Huiqi Qu has a background as a physician trained in internal medicine and a PhD in Experimental Medicine (Endocrinology). His research applies human genetics and multi-omics to complex and pediatric diseases, including GWAS, polygenic risk scores (PRS), single-cell transcriptomics, and integrative genomics to support precision medicine and clinical translation.\",\"sameAs\":[\"https:\\\/\\\/x.com\\\/HuiQiQu\"],\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/author\\\/hqiqu\\\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/","og_locale":"en_US","og_type":"article","og_title":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog","og_description":"Retinal diseases can result from genetic mutations or combined risk factors, all causing vision impairment progressing to blindness. To date, available therapeutic approaches result in inefficient and expensive treatments. The CRISPR-Cas9 system, an RNA-guided genome editing technology, may be applied to precisely correct or remove disease-related genetic mutations, or induce permanent inhibition of factors promoting [...]Read More...","og_url":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/","og_site_name":"JMG Contact blog","article_published_time":"2019-12-20T15:42:34+00:00","article_modified_time":"2026-02-24T00:20:41+00:00","author":"hqqu","twitter_card":"summary_large_image","twitter_creator":"@HuiQiQu","twitter_misc":{"Written by":"hqqu","Est. reading time":"1 minute"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/#article","isPartOf":{"@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/"},"author":{"name":"hqqu","@id":"https:\/\/blogs.bmj.com\/jmg\/#\/schema\/person\/be0250f8d5b52412c3e7c222dabd591b"},"headline":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia)","datePublished":"2019-12-20T15:42:34+00:00","dateModified":"2026-02-24T00:20:41+00:00","mainEntityOfPage":{"@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/"},"wordCount":128,"commentCount":0,"publisher":{"@id":"https:\/\/blogs.bmj.com\/jmg\/#organization"},"inLanguage":"en-US","potentialAction":[{"@type":"CommentAction","name":"Comment","target":["https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/#respond"]}]},{"@type":"WebPage","@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/","url":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/","name":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia) - JMG Contact blog","isPartOf":{"@id":"https:\/\/blogs.bmj.com\/jmg\/#website"},"datePublished":"2019-12-20T15:42:34+00:00","dateModified":"2026-02-24T00:20:41+00:00","breadcrumb":{"@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/blogs.bmj.com\/jmg\/2019\/12\/20\/gene-editing-prospects-for-treating-inherited-retinal-diseases\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/blogs.bmj.com\/jmg\/"},{"@type":"ListItem","position":2,"name":"Gene editing prospects for treating inherited retinal diseases (Contributed by Dr. Alessandra Recchia)"}]},{"@type":"WebSite","@id":"https:\/\/blogs.bmj.com\/jmg\/#website","url":"https:\/\/blogs.bmj.com\/jmg\/","name":"JMG Contact blog","description":"JMG Contact blog","publisher":{"@id":"https:\/\/blogs.bmj.com\/jmg\/#organization"},"potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/blogs.bmj.com\/jmg\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"},{"@type":"Organization","@id":"https:\/\/blogs.bmj.com\/jmg\/#organization","name":"JMG Contact blog","url":"https:\/\/blogs.bmj.com\/jmg\/","logo":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/blogs.bmj.com\/jmg\/#\/schema\/logo\/image\/","url":"https:\/\/blogs.bmj.com\/jmg\/files\/2017\/10\/blog-logo-jmg.png","contentUrl":"https:\/\/blogs.bmj.com\/jmg\/files\/2017\/10\/blog-logo-jmg.png","width":300,"height":34,"caption":"JMG Contact blog"},"image":{"@id":"https:\/\/blogs.bmj.com\/jmg\/#\/schema\/logo\/image\/"}},{"@type":"Person","@id":"https:\/\/blogs.bmj.com\/jmg\/#\/schema\/person\/be0250f8d5b52412c3e7c222dabd591b","name":"hqqu","image":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/secure.gravatar.com\/avatar\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g","url":"https:\/\/secure.gravatar.com\/avatar\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g","contentUrl":"https:\/\/secure.gravatar.com\/avatar\/920bc4e81500cde88def4bfd7775029cb154e422c5460b9cf80d5c47137d6f35?s=96&d=mm&r=g","caption":"hqqu"},"description":"Huiqi Qu has a background as a physician trained in internal medicine and a PhD in Experimental Medicine (Endocrinology). His research applies human genetics and multi-omics to complex and pediatric diseases, including GWAS, polygenic risk scores (PRS), single-cell transcriptomics, and integrative genomics to support precision medicine and clinical translation.","sameAs":["https:\/\/x.com\/HuiQiQu"],"url":"https:\/\/blogs.bmj.com\/jmg\/author\/hqiqu\/"}]}},"_links":{"self":[{"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/posts\/1396","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/users\/123"}],"replies":[{"embeddable":true,"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/comments?post=1396"}],"version-history":[{"count":0,"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/posts\/1396\/revisions"}],"wp:attachment":[{"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/media?parent=1396"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/categories?post=1396"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/blogs.bmj.com\/jmg\/wp-json\/wp\/v2\/tags?post=1396"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}