1.0Revista Brasileira de Oftalmologiahttps://www.rbojournal.org/en/rich600338<blockquote class="wp-embedded-content" data-secret="anJGgYfu7R"><a href="https://www.rbojournal.org/en/article/gene-therapy-for-retinal-diseases/">Gene therapy for retinal diseases</a></blockquote><iframe sandbox="allow-scripts" security="restricted" src="https://www.rbojournal.org/en/article/gene-therapy-for-retinal-diseases/embed/#?secret=anJGgYfu7R" width="600" height="338" title="“Gene therapy for retinal diseases” — Revista Brasileira de Oftalmologia" data-secret="anJGgYfu7R" frameborder="0" marginwidth="0" marginheight="0" scrolling="no" class="wp-embedded-content"></iframe><script type="text/javascript"> /* <![CDATA[ */ /*! This file is auto-generated */ !function(d,l){"use strict";l.querySelector&&d.addEventListener&&"undefined"!=typeof URL&&(d.wp=d.wp||{},d.wp.receiveEmbedMessage||(d.wp.receiveEmbedMessage=function(e){var t=e.data;if((t||t.secret||t.message||t.value)&&!/[^a-zA-Z0-9]/.test(t.secret)){for(var s,r,n,a=l.querySelectorAll('iframe[data-secret="'+t.secret+'"]'),o=l.querySelectorAll('blockquote[data-secret="'+t.secret+'"]'),c=new RegExp("^https?:$","i"),i=0;i<o.length;i++)o[i].style.display="none";for(i=0;i<a.length;i++)s=a[i],e.source===s.contentWindow&&(s.removeAttribute("style"),"height"===t.message?(1e3<(r=parseInt(t.value,10))?r=1e3:~~r<200&&(r=200),s.height=r):"link"===t.message&&(r=new URL(s.getAttribute("src")),n=new URL(t.value),c.test(n.protocol))&&n.host===r.host&&l.activeElement===s&&(d.top.location.href=t.value))}},d.addEventListener("message",d.wp.receiveEmbedMessage,!1),l.addEventListener("DOMContentLoaded",function(){for(var e,t,s=l.querySelectorAll("iframe.wp-embedded-content"),r=0;r<s.length;r++)(t=(e=s[r]).getAttribute("data-secret"))||(t=Math.random().toString(36).substring(2,12),e.src+="#?secret="+t,e.setAttribute("data-secret",t)),e.contentWindow.postMessage({message:"ready",secret:t},"*")},!1)))}(window,document); //# sourceURL=https://www.rbojournal.org/wp-includes/js/wp-embed.min.js /* ]]> */ </script> ABSTRACT This update article aims to discuss gene therapies under study for retinal diseases, mainly among the different inherited retinal dystrophies. Inherited retinal dystrophies are considered rare diseases, some are ultra-rare. The use of Luxturna, which was approved by the FDA in 2017 and by Anvisa in 2020, changed the natural history of the retinopathy associated with biallelic variants in RPE65 gene and was a major milestone in ophthalmology. There are still many challenges for this new therapeutic modality, which [...]