{"id":189,"date":"2024-08-14T14:06:01","date_gmt":"2024-08-14T14:06:01","guid":{"rendered":"https:\/\/stgbeyond9.wpenginepowered.com\/?page_id=189"},"modified":"2026-01-22T12:47:38","modified_gmt":"2026-01-22T12:47:38","slug":"what-is-gene-therapy","status":"publish","type":"page","link":"https:\/\/hemophilia-beyond-9.com\/en-au\/what-is-gene-therapy\/","title":{"rendered":"What is gene therapy?"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"189\" class=\"elementor elementor-189\" data-elementor-post-type=\"page\">\n\t\t\t\t<div class=\"elementor-element elementor-element-1ff6fb5 header-background e-flex e-con-boxed e-con e-parent\" data-id=\"1ff6fb5\" data-element_type=\"container\" data-e-type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-2c3a458 e-con-full e-flex e-con e-child\" data-id=\"2c3a458\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-176ab3a elementor-widget__width-initial newie elementor-widget elementor-widget-heading\" data-id=\"176ab3a\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h1 class=\"elementor-heading-title elementor-size-default\">What is gene insertion therapy and CRISPR technology? <\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-80742d6 e-con-full e-flex e-con e-child\" data-id=\"80742d6\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-671f5ff e-flex e-con-boxed e-con e-parent\" data-id=\"671f5ff\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t\t\t<div class=\"elementor-element elementor-element-63aff7e elementor-widget elementor-widget-text-editor\" data-id=\"63aff7e\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\tCRISPR stands for \u201cclustered regularly interspaced short palindromic repeats.\u201d Cas9 is a                    CRISPR-associated protein 9. CRISPR technology has two parts which work together for gene insertion: a\u202fguide RNA strand and a Cas9 enzyme. The guide RNA strand guides the Cas9\u202fenzyme precisely to a matching location within the DNA sequence. Only then\u202fwill the Cas9 enzyme create a small opening for the new gene to be inserted. \n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-47fd7ad background-left e-flex e-con-boxed e-con e-parent\" data-id=\"47fd7ad\" data-element_type=\"container\" data-e-type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-27d0a6c e-con-full e-flex e-con e-child\" data-id=\"27d0a6c\" data-element_type=\"container\" data-e-type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t<div class=\"elementor-element elementor-element-49eaf45 elementor-widget elementor-widget-image\" data-id=\"49eaf45\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img fetchpriority=\"high\" decoding=\"async\" width=\"470\" height=\"473\" src=\"https:\/\/hemophilia-beyond-9.com\/wp-content\/uploads\/2024\/08\/GT2.svg\" class=\"attachment-full size-full wp-image-266\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-40cb865 e-con-full e-flex e-con e-child\" data-id=\"40cb865\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-50d84e5 elementor-widget elementor-widget-text-editor\" data-id=\"50d84e5\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\tThe investigational treatment is a CRISPR-based targeted gene insertion therapy which works in two steps, which are both required for the therapy to work. The first step involves delivery where a viral vector delivers a therapeutic gene into a target liver cell, while a lipid-based carrier delivers the CRISPR technology to the target gene. The second step involves targeted insertion, where once inside the liver cell, CRISPR technology targets a precise location within the DNA, where the new, therapeutic gene is inserted. Together, the two steps are called CRISPR gene insertion. CRISPR gene insertion adds the new gene directly into the DNA at a precise location so that it becomes a permanent part of your DNA. This is different from some other gene therapies that involve a virus bringing a new gene into a cell that is not intended to become part of the DNA of the cell. \n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-e219853 elementor-widget elementor-widget-text-editor\" data-id=\"e219853\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\tAt this time, there are no approved therapies that are based on using CRISPR gene insertion. However, CRISPR technology is being studied in other clinical trials to treat other genetic diseases.\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-c2f9c52 e-flex e-con-boxed e-con e-parent\" data-id=\"c2f9c52\" data-element_type=\"container\" data-e-type=\"container\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t\t\t<div class=\"elementor-element elementor-element-13e0c7a elementor-widget elementor-widget-video\" data-id=\"13e0c7a\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;video_type&quot;:&quot;hosted&quot;,&quot;controls&quot;:&quot;yes&quot;}\" data-widget_type=\"video.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"e-hosted-video elementor-wrapper elementor-open-inline\">\n\t\t\t\t\t<video class=\"elementor-video\" src=\"\/wp-content\/uploads\/2025\/08\/[Regeneron] - [Beyond9Study  - 27-11-2024 - English (Principal) - 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CRISPR stands for \u201cclustered regularly interspaced short palindromic repeats.\u201d Cas9 is a CRISPR-associated protein 9. CRISPR technology has two parts which work together for gene insertion: a\u202fguide RNA strand and a Cas9 enzyme. The guide RNA strand guides the Cas9\u202fenzyme precisely to a matching location within the [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-189","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/pages\/189","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/comments?post=189"}],"version-history":[{"count":0,"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/pages\/189\/revisions"}],"wp:attachment":[{"href":"https:\/\/hemophilia-beyond-9.com\/en-au\/wp-json\/wp\/v2\/media?parent=189"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}