640 lines
30 KiB
Text
640 lines
30 KiB
Text
|
||
<!DOCTYPE html>
|
||
<html lang="en" dir="ltr">
|
||
<head>
|
||
<meta charset="utf-8" />
|
||
<meta name="description" content="Scientists at the University of Wisconsin‒Madison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable." />
|
||
<meta name="keywords" content="Genetics,Rare Diseases,Regenerative Medicine,Gene Therapy,Retina" />
|
||
<meta name="google" content="vityevxlt8UuVFWvgz9_YwmJ5F0Nby_B3t54LLh-3Zw,lACaj571Gx_VdLF3HKSKUf1Wxx0H_bWxO3BYDZ_C1uw" />
|
||
<meta name="google-site-verification" content="vityevxlt8UuVFWvgz9_YwmJ5F0Nby_B3t54LLh-3Zw" />
|
||
<meta name="google-site-verification" content="lACaj571Gx_VdLF3HKSKUf1Wxx0H_bWxO3BYDZ_C1uw" />
|
||
<meta name="google-site-verification" content="UDfyeEAWNX_rJUHwog9j1kziEnByGwhkSJGLJclUWDA" />
|
||
<meta name="viewport" content="width=device-width, initial-scale=1.0" />
|
||
<script type="application/ld+json">{
|
||
"@context": "https://schema.org",
|
||
"@graph": [
|
||
{
|
||
"@type": "NewsArticle",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness",
|
||
"name": "UW researchers devise approach to treat rare, incurable form of blindness",
|
||
"headline": "UW researchers devise approach to treat rare, incurable form of blindness",
|
||
"image": {
|
||
"@type": "ImageObject",
|
||
"representativeOfPage": "False",
|
||
"url": "https://www.nei.nih.gov/sites/default/files/styles/medium/public/2020-08/David-Gamm-lab-800x533.jpg?itok=qQfQRlCH",
|
||
"width": "800",
|
||
"height": "533"
|
||
},
|
||
"datePublished": "2020-07-30T10:46:11-0400",
|
||
"dateModified": "2025-01-24T14:52:23-0500",
|
||
"author": {
|
||
"@type": "GovernmentOrganization",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness",
|
||
"name": "National Eye Institute",
|
||
"url": "https://www.nei.nih.gov"
|
||
},
|
||
"publisher": {
|
||
"@type": "GovernmentOrganization",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness",
|
||
"name": "National Eye Institute",
|
||
"url": "https://www.nei.nih.gov"
|
||
}
|
||
},
|
||
{
|
||
"@type": "WebPage",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness",
|
||
"breadcrumb": {
|
||
"@type": "BreadcrumbList",
|
||
"itemListElement": [
|
||
{
|
||
"@type": "ListItem",
|
||
"position": 1,
|
||
"name": "Home",
|
||
"item": "https://www.nei.nih.gov/"
|
||
},
|
||
{
|
||
"@type": "ListItem",
|
||
"position": 2,
|
||
"name": "About NEI",
|
||
"item": "https://www.nei.nih.gov/about"
|
||
},
|
||
{
|
||
"@type": "ListItem",
|
||
"position": 3,
|
||
"name": "News and Events",
|
||
"item": "https://www.nei.nih.gov/about/news-and-events"
|
||
},
|
||
{
|
||
"@type": "ListItem",
|
||
"position": 4,
|
||
"name": "NEI Research News",
|
||
"item": "https://www.nei.nih.gov/about/news-and-events/news"
|
||
},
|
||
{
|
||
"@type": "ListItem",
|
||
"position": 5,
|
||
"name": "UW researchers devise approach to treat rare, incurable form of blindness",
|
||
"item": "https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness"
|
||
}
|
||
]
|
||
},
|
||
"author": {
|
||
"@type": "GovernmentOrganization",
|
||
"@id": "https://www.nei.nih.gov",
|
||
"name": "National Eye Institute",
|
||
"url": "https://www.nei.nih.gov"
|
||
},
|
||
"publisher": {
|
||
"@type": "GovernmentOrganization",
|
||
"@id": "https://www.nei.nih.gov",
|
||
"name": "National Eye Institute",
|
||
"url": "https://www.nei.nih.gov"
|
||
}
|
||
}
|
||
]
|
||
}</script>
|
||
<link rel="icon" type="image/png" href="/themes/custom/nei/images/favicon.png" />
|
||
<link rel="apple-touch-icon" href="/themes/custom/nei/images/apple-touch-icon.png" />
|
||
<meta name="last-updated" content="Jan 24, 2025, 2:52:23 PM" />
|
||
<link rel="icon" href="/themes/custom/nei/favicon.ico" type="image/vnd.microsoft.icon" />
|
||
<link rel="alternate" hreflang="en" href="https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness" />
|
||
<link rel="canonical" href="https://www.nei.nih.gov/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness" />
|
||
<link rel="shortlink" href="https://www.nei.nih.gov/node/3153" />
|
||
|
||
<title>UW researchers devise approach to treat rare, incurable form of blindness | National Eye Institute</title>
|
||
<link rel="preload" href="/themes/custom/nei/fonts/Aleo-Bold.woff2" as="font" type="font/woff2" crossorigin>
|
||
<link rel="preload" href="/themes/custom/nei/fonts/Lato-Regular.woff2" as="font" type="font/woff2" crossorigin>
|
||
<link rel="stylesheet" media="all" href="/sites/default/files/css/css_PGSk_7b88hq2qr_--_38MLvoj1ffr9jv3JhnNJTnKUM.css?delta=0&language=en&theme=nei&include=eJw1ytEKgCAMRuEXsnwk-dMxrOliTqi3j4iuDnycfFCprpbyHK4tDb-FRvwSshrFYvOErNhxBUHnCaYEcbJ_61Qji26Q5ZXa-QH_YCJ9" />
|
||
<link rel="stylesheet" media="all" href="/themes/custom/nei/css/style.css?sraguf" />
|
||
|
||
<script type="application/json" data-drupal-selector="drupal-settings-json">{"path":{"baseUrl":"\/","pathPrefix":"","currentPath":"node\/3153","currentPathIsAdmin":false,"isFront":false,"currentLanguage":"en"},"pluralDelimiter":"\u0003","gtag":{"tagId":"","consentMode":false,"otherIds":[],"events":[],"additionalConfigInfo":[]},"suppressDeprecationErrors":true,"ajaxPageState":{"libraries":"eJxdjFsOwjAMBC8U6iNFbmKZgPNQ7BTK6SmgCsrPrndkTbhSTFa7D0OtZq-2Cil8yoWOj5WYYT8c18pC3pCBt_jfE17wfoTZCRYeyORRjPruLmhpIS-b2EvFmArD73CFErDUGeWkoadmekCb5fXVsCN3bGeF2EdDmb5kGqWNWZKeKTpd1SjDjEpuSXRTeOeUaxxCT9WYZ50","theme":"nei","theme_token":null},"ajaxTrustedUrl":{"form_action_p_pvdeGsVG5zNF_XLGPTvYSKCf43t8qZYSwcfZl2uzM":true},"gtm":{"tagId":null,"settings":{"data_layer":"dataLayer","include_classes":false,"allowlist_classes":"google\nnonGooglePixels\nnonGoogleScripts\nnonGoogleIframes","blocklist_classes":"customScripts\ncustomPixels","include_environment":false,"environment_id":"","environment_token":""},"tagIds":["GTM-TLVNS7W"]},"user":{"uid":0,"permissionsHash":"c48594e108b5c1c2bea409821b71477d5bc2724c8cae6d31f1d985af0daab85c"}}</script>
|
||
<script src="/sites/default/files/js/js_WgzMsgqb-MiW-Z2aLffGQHepZwX2hWrEHSrb46WqZrk.js?scope=header&delta=0&language=en&theme=nei&include=eJxdjEEOgCAMBD-E9kmkYrOpqdQAMerr5eBBvUx29jCp8HUKQOkZAe4wiY1B6Pj7yAsf33MNmZvuEq0nojnPmkFvCVmUYD6xDTUV3Vq9ATUbLxk"></script>
|
||
<script src="https://script.crazyegg.com/pages/scripts/0117/6571.js" async></script>
|
||
<script src="/modules/contrib/google_tag/js/gtag.js?sraguf"></script>
|
||
<script src="/modules/contrib/google_tag/js/gtm.js?sraguf"></script>
|
||
|
||
</head>
|
||
<body class="t-news_item t-node__3153 path-about-news-and-events-news-uw-researchers-devise-approach-treat-rare-incurable-form-blindness">
|
||
<a href="#main-content" class="u-skip-link">
|
||
Skip to content
|
||
</a>
|
||
<noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-TLVNS7W"
|
||
height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript>
|
||
|
||
<div class="dialog-off-canvas-main-canvas" data-off-canvas-main-canvas>
|
||
|
||
<div id="u-page-outer" class="u-outer">
|
||
<div class="views-element-container site-notice-block" id="block-views-block-site-notices-block-1">
|
||
|
||
|
||
|
||
|
||
|
||
<div>
|
||
<div class="js-view-dom-id-bca3892fe522a94248c74087cbf8646ffbc018befc8d0e64978ba8c06dc01631 site-notices">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
</div>
|
||
</div>
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
<img src="/themes/custom/nei/images/NEI-logo-tagline.svg" alt="" width="200" height="48" class="u-print-only">
|
||
<div class="r-global-header">
|
||
<div id="r-header-outer" class="r-header">
|
||
|
||
<div class="r-header-inner">
|
||
|
||
<div id="block-nei-branding" class="o-block--logo" role="banner">
|
||
|
||
|
||
|
||
|
||
|
||
<div class="o-logo">
|
||
<a href="/" class="o-logo__link" title="Home" rel="home"><span class="o-logo__text">National Eye Institute</span> <span class="o-logo__tagline">Research Today… Vision Tomorrow</span></a>
|
||
</div>
|
||
|
||
|
||
</div>
|
||
|
||
<div id="block-neimenubutton-2">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<div class="o-menu-toggle"><button class="o-menu-toggle__btn" id="menu-toggle__btn" aria-controls="block-mainnavigation" aria-expanded="true">Menu</button></div>
|
||
|
||
|
||
|
||
|
||
</div>
|
||
<div class="language-switcher-language-url has-no-translation" id="block-languageswitcher" role="navigation" aria-label="Language switcher">
|
||
|
||
|
||
<ul class="links">
|
||
<li class="is-active">
|
||
<a href="/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness" class="language-link is-active" lang="en" data-drupal-link-system-path="node/3153" aria-current="page">English</a>
|
||
</li>
|
||
<li>
|
||
<a href="/espanol/node/3153" class="language-link is-active" lang="es" data-drupal-link-system-path="node/3153" aria-current="page"> Español</a>
|
||
</li>
|
||
</ul>
|
||
|
||
|
||
</div>
|
||
<div class="search-global c-search--global" data-drupal-selector="search-global" id="block-globalsearchform" role="search">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<form action="/about/news-and-events/news/uw-researchers-devise-approach-treat-rare-incurable-form-blindness" method="post" id="search-global" accept-charset="UTF-8">
|
||
<div class="c-form__inner">
|
||
<div class="js-form-item form-item js-form-type-textfield form-item-global-terms js-form-item-global-terms">
|
||
<label for="edit-global-terms">Search the site</label>
|
||
<input data-drupal-selector="edit-global-terms" type="text" id="edit-global-terms" name="global-terms" value="" size="30" maxlength="128" placeholder="Search the site" class="form-text" />
|
||
|
||
</div>
|
||
<input data-drupal-selector="form-wj-zmzz8yiid5xoy-roxwnszlzi0hmuf17f-fhyqq5u" type="hidden" name="form_build_id" value="form-wj-zMzz8YiID5xOy_roxWnSzLzI0HMUf17f-fhyQQ5U" />
|
||
<input data-drupal-selector="edit-search-global" type="hidden" name="form_id" value="search_global" />
|
||
<div data-drupal-selector="edit-global-actions" class="form-actions js-form-wrapper form-wrapper" id="edit-global-actions"><input data-drupal-selector="edit-global-submit" type="submit" id="edit-global-submit" name="op" value="Search" class="button js-form-submit form-submit" />
|
||
</div>
|
||
|
||
</div>
|
||
</form>
|
||
|
||
|
||
|
||
|
||
|
||
</div>
|
||
|
||
</div>
|
||
|
||
</div>
|
||
|
||
<div class="r-menu-primary">
|
||
<nav aria-label="Main navigation" id="block-mainnavigation" class="o-menu-main__outer">
|
||
|
||
<ul class="o-menu o-menu-main"> <li class="o-menu-main__item o-menu-main__item--expanded o-menu-main__item--no-js"> <a href="/learn-about-eye-health" class="o-menu-main__link" data-drupal-link-system-path="node/39">Learn About Eye Health</a>
|
||
<ul class="o-menu o-menu-main__submenu"> <li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/learn-about-eye-health/healthy-vision" class="o-menu-main__link" data-drupal-link-system-path="node/788">Healthy Vision</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/learn-about-eye-health/eye-conditions-and-diseases" class="o-menu-main__link" data-drupal-link-system-path="learn-about-eye-health/eye-conditions-and-diseases">Eye Conditions and Diseases</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/learn-about-eye-health/eye-health-data-and-statistics" class="o-menu-main__link" data-drupal-link-system-path="node/825">Eye Health Data and Statistics</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/learn-about-eye-health/campaigns" class="o-menu-main__link" data-drupal-link-system-path="node/3859">Campaigns</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/learn-about-eye-health/vision-rehabilitation" class="o-menu-main__link" data-drupal-link-system-path="node/3865">Vision Rehabilitation</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/learn-about-eye-health/nei-for-kids" class="o-menu-main__link" data-drupal-link-system-path="node/162">NEI for Kids</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/learn-about-eye-health/outreach-resources" class="o-menu-main__link" data-drupal-link-system-path="node/125">Outreach Resources</a>
|
||
</li>
|
||
</ul>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--expanded o-menu-main__item--no-js"> <a href="/grants-and-training" class="o-menu-main__link" data-drupal-link-system-path="node/143">Grants and Training</a>
|
||
<ul class="o-menu-main__submenu o-menu"> <li class="o-menu-main__item"> <a href="/grants-and-training/how-apply" class="o-menu-main__link" data-drupal-link-system-path="node/145">How to Apply</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/funding-opportunities" class="o-menu-main__link" data-drupal-link-system-path="node/144">Funding Opportunities</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/training-nei" class="o-menu-main__link" data-drupal-link-system-path="node/146">Training at NEI</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/funding-training-and-career-development" class="o-menu-main__link" data-drupal-link-system-path="node/152">Funding for Training and Career Development</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/policies-and-procedures" class="o-menu-main__link" data-drupal-link-system-path="node/149">Policies and Procedures</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/grants-and-training/Prior-Approval-Requests" class="o-menu-main__link" data-drupal-link-system-path="node/3918">Prior Approval Requests</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/resources-researchers" class="o-menu-main__link" data-drupal-link-system-path="node/150">Resources for Researchers</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/grants-and-training/contact-grants-and-funding-staff" class="o-menu-main__link" data-drupal-link-system-path="node/151">Contact Grants and Funding Staff</a>
|
||
</li>
|
||
</ul>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--expanded o-menu-main__item--no-js"> <a href="/research" class="o-menu-main__link" data-drupal-link-system-path="node/41">Research at NEI</a>
|
||
<ul class="o-menu-main__submenu o-menu"> <li class="o-menu-main__item"> <a href="/about/news-and-events/news" title="Research News" class="o-menu-main__link" data-drupal-link-system-path="about/news-and-events/news">Research News</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/research/clinical-trials" class="o-menu-main__link" data-drupal-link-system-path="node/132">Clinical Trials</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/research/research-labs-and-branches" class="o-menu-main__link" data-drupal-link-system-path="node/270">Research Labs and Branches</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/research/jobs-and-training-opportunities-nei" class="o-menu-main__link" data-drupal-link-system-path="node/564">Jobs and Training Opportunities at NEI</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/research/seminars" class="o-menu-main__link" data-drupal-link-system-path="node/4048">NEI Research Seminars</a>
|
||
</li>
|
||
</ul>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--expanded o-menu-main__item--no-js"> <a href="/about" class="o-menu-main__link" data-drupal-link-system-path="node/275">About NEI</a>
|
||
<ul class="o-menu-main__submenu o-menu"> <li class="o-menu-main__item"> <a href="/about/strategic-planning" class="o-menu-main__link" data-drupal-link-system-path="node/2857">Strategic Planning</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/news-and-events" class="o-menu-main__link" data-drupal-link-system-path="node/131">News and Events</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/goals-and-accomplishments" class="o-menu-main__link" data-drupal-link-system-path="node/281">Goals and Accomplishments</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/nei-history" class="o-menu-main__link" data-drupal-link-system-path="node/282">NEI History</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/nei-leadership" class="o-menu-main__link" data-drupal-link-system-path="node/415">NEI Leadership</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/budget-and-congress" class="o-menu-main__link" data-drupal-link-system-path="node/395">Budget and Congress</a>
|
||
</li>
|
||
<li class="o-menu-main__item o-menu-main__item--collapsed"> <a href="/about/advisory-committees" class="o-menu-main__link" data-drupal-link-system-path="node/285">Advisory Committees</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/learn-about-eye-health/outreach-resources/national-eye-health-education-program" title="National Eye Health Education Program" class="o-menu-main__link" data-drupal-link-system-path="node/265">National Eye Health Education Program</a>
|
||
</li>
|
||
<li class="o-menu-main__item"> <a href="/about/donate-nei" class="o-menu-main__link" data-drupal-link-system-path="node/286">Donate to NEI</a>
|
||
</li>
|
||
</ul>
|
||
</li>
|
||
</ul>
|
||
|
||
|
||
</nav>
|
||
|
||
|
||
</div>
|
||
</div>
|
||
|
||
<div class="o-overlay" aria-hidden="true"></div>
|
||
|
||
<div class="r-breadcrumb">
|
||
|
||
<div id="block-nei-breadcrumbs">
|
||
|
||
|
||
|
||
|
||
|
||
<nav aria-label="Breadcrumb">
|
||
<ol class="c-breadcrumb">
|
||
<li>
|
||
<a href="/">Home</a>
|
||
</li>
|
||
<li>
|
||
<a href="/about">About NEI</a>
|
||
</li>
|
||
<li>
|
||
<a href="/about/news-and-events">News and Events</a>
|
||
</li>
|
||
<li>
|
||
<a href="/about/news-and-events/news">NEI Research News</a>
|
||
</li>
|
||
<li>
|
||
UW researchers devise approach to treat rare, incurable form of blindness
|
||
</li>
|
||
</ol>
|
||
</nav>
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<div class="r-page-header">
|
||
<div class="o-page-header">
|
||
|
||
<div id="block-nei-page-title">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<h1>
|
||
<span>UW researchers devise approach to treat rare, incurable form of blindness</span>
|
||
</h1>
|
||
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
|
||
|
||
</div>
|
||
</div>
|
||
|
||
<div class="r-highlighted">
|
||
<div data-drupal-messages-fallback class="hidden"></div>
|
||
|
||
|
||
</div>
|
||
|
||
<div class="r-help">
|
||
|
||
</div>
|
||
<main id="main-content" class="r-main">
|
||
<div class="r-pre-content">
|
||
|
||
</div>
|
||
|
||
<div class="r-splitter">
|
||
<div class="r-content">
|
||
|
||
<div id="block-nei-content">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<article data-history-node-id="3153">
|
||
|
||
|
||
|
||
<div>
|
||
|
||
|
||
|
||
|
||
<div class="c-item__subtitle">
|
||
Scientists at the University of Wisconsin‒Madison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.
|
||
</div>
|
||
|
||
|
||
<div class="c-item__date">
|
||
July 30, 2020
|
||
</div>
|
||
|
||
|
||
|
||
|
||
<div class="c-item__tag c-item__tag--topic">
|
||
<a href="/about/news-and-events/news?topic=401" hreflang="en">Gene Therapy</a>
|
||
<a href="/about/news-and-events/news?topic=402" hreflang="en">Genetics</a>
|
||
<a href="/about/news-and-events/news?topic=56" hreflang="en">Rare Diseases</a>
|
||
<a href="/about/news-and-events/news?topic=364" hreflang="en">Regenerative Medicine</a>
|
||
<a href="/about/news-and-events/news?topic=7" hreflang="en">Retina</a>
|
||
</div>
|
||
|
||
|
||
|
||
|
||
|
||
<div class="c-item__tag c-item__tag--research-type">
|
||
<a href="/about/news-and-events/news?research=35" hreflang="en">Translational Research</a>
|
||
</div>
|
||
|
||
|
||
|
||
|
||
|
||
<div class="c-item__tag c-item__tag--source">
|
||
<a href="/about/news-and-events/news?source=29" hreflang="en">Grantee</a>
|
||
</div>
|
||
|
||
|
||
|
||
|
||
|
||
<div class="o-news__content">
|
||
|
||
<div class="c-image c-image--left">
|
||
<div class="c-image__src">
|
||
<img src="https://www.nei.nih.gov/sites/default/files/styles/small/public/2020-08/David-Gamm-lab-800x533.jpg?itok=3ANeisqY" alt="David Gamm in the laboratory" width="800" height="533">
|
||
</div>
|
||
</div>
|
||
|
||
<div class="c-text">
|
||
|
||
|
||
|
||
|
||
<p>Best vitelliform macular degeneration, or Best disease, is an inherited eye condition that typically leads to blindness over the course of a few decades. The disease can be caused by more than two hundred mutations in the <em>BEST1</em> gene.</p><p>The researchers were able to correct the disease in stem cells from patients with <em>BEST1</em> mutations by overwhelming broken copies of the gene with many functional copies of <em>BEST1</em>. This approach worked for most, but not all, of the <em>BEST1</em> mutations that they tested. As an alternative approach for mutations that did not respond to this gene augmentation method, the team used CRISPR-Cas9 gene editing to target and correct the mutations.</p>
|
||
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<div class="c-link">
|
||
|
||
<a href="https://www.med.wisc.edu/news-and-events/2020/july/researchers-devise-new-treatment-for-best-disease/" class="c-link__link">Read more at University of Wisconsin-Madison School of Medicine and Public Health News</a>
|
||
|
||
</div>
|
||
|
||
</div>
|
||
|
||
|
||
</div>
|
||
|
||
</article>
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
</div>
|
||
<aside class="r-sidebar" role="complementary">
|
||
|
||
<div class="views-element-container" id="block-views-block-nei-sidebar-block-1">
|
||
|
||
|
||
|
||
|
||
|
||
<div>
|
||
<div class="js-view-dom-id-f22b5ab95786145511216793cbb52268aa05857e0c893506c78a28f8dcf4b91b nei-sidebar">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
</div>
|
||
</div>
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
</aside>
|
||
</div>
|
||
</main>
|
||
|
||
<div class="r-footer">
|
||
<footer class="o-footer" role="contentinfo">
|
||
|
||
<div id="block-footer-2">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<div class="o-footer__row">
|
||
<div class="o-footer__group o-footer__group--logo"><a class="o-logo__link" href="/" rel="home" title="Home"><span class="o-logo__text">National Eye Institute</span></a>
|
||
<ul class="o-menu-social">
|
||
<li class="o-menu-social__item"><a class="o-menu-social__link o-menu-social__link--facebook outlink-ignore" href="https://www.facebook.com/NationalEyeInstitute">Facebook</a></li>
|
||
<li class="o-menu-social__item"><a class="o-menu-social__link o-menu-social__link--twitter outlink-ignore" href="https://twitter.com/NatEyeInstitute">Twitter</a></li>
|
||
<li class="o-menu-social__item"><a class="o-menu-social__link o-menu-social__link--linkedin outlink-ignore" href="https://www.linkedin.com/company/national-eye-institute-nei">LinkedIn</a></li>
|
||
<li class="o-menu-social__item"><a class="o-menu-social__link o-menu-social__link--youtube outlink-ignore" href="https://www.youtube.com/user/neinih">YouTube</a></li>
|
||
</ul>
|
||
|
||
<p> </p>
|
||
</div>
|
||
|
||
<div class="o-footer__group o-footer__group--contact">
|
||
<h2 class="o-footer__title">Contact NEI</h2>
|
||
|
||
<ul class="o-menu-footer">
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/contact-us/">Contact Us</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://www.nih.gov/about-nih/visitor-information">Visit NIH</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://list.nih.gov/cgi-bin/wa.exe?SUBED1=insidenei&A=1">Get Email Updates from NEI</a></li>
|
||
</ul>
|
||
</div>
|
||
|
||
<div class="o-footer__group o-footer__group--policies">
|
||
<h2 class="o-footer__title">Policies</h2>
|
||
|
||
<ul class="o-menu-footer">
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/accessibility-information">Accessibility</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://www.nih.gov/nondiscrimination-notice-language-assistance-services">Information in Other Languages</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/nei-website-privacy-policy">Privacy</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/freedom-information-act">Freedom of Information Act (FOIA)</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/disclaimers">Disclaimers</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="/web-policies ">Web Policies</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html ">Vulnerability Disclosure</a></li>
|
||
</ul>
|
||
</div>
|
||
|
||
<div class="o-footer__group o-footer__group--employees">
|
||
<h2 class="o-footer__title">For NEI Employees</h2>
|
||
|
||
<ul class="o-menu-footer">
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://intranet.nei.nih.gov">Intranet</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://intranet.nei.nih.gov/hr/Pages/emergency.aspx">Emergency Information</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://ned.nih.gov/search/search.aspx">NIH Staff Directory</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
|
||
<div class="o-footer__row">
|
||
<div class="o-footer__full o-footer__full--parent">
|
||
<ul class="o-menu-footer o-menu-footer--swing">
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://www.nih.gov/">NIH.gov</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://hhs.gov/">HHS.gov</a></li>
|
||
<li class="o-menu-footer__item"><a class="o-menu-footer__link" href="https://usa.gov/">USA.gov</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
|
||
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
</footer>
|
||
</div>
|
||
|
||
</div>
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<div class="c-lb-wrapper" id="lb-wrapper" aria-hidden="true">
|
||
<div class="c-lb-overlay" tabindex="-1"></div>
|
||
<div role="dialog" class="c-lb" id="lb">
|
||
<div class="c-lb-header">
|
||
<button class="c-lb-close-btn"><span class="c-lb-close-btn-text">Close</span><span class="c-lb-close-btn-text times" aria-hidden="true">×</span></button>
|
||
</div>
|
||
<figure class="c-lb-figure">
|
||
<figcaption class="c-lb-caption"></figcaption>
|
||
</figure>
|
||
</div>
|
||
</div>
|
||
|
||
<script src="/sites/default/files/js/js_agazM-qm5cOcplNZo9kNIWFtUrwt0V1xgAKtLyM44Us.js?scope=footer&delta=0&language=en&theme=nei&include=eJxdjEEOgCAMBD-E9kmkYrOpqdQAMerr5eBBvUx29jCp8HUKQOkZAe4wiY1B6Pj7yAsf33MNmZvuEq0nojnPmkFvCVmUYD6xDTUV3Vq9ATUbLxk"></script>
|
||
<script src="/themes/custom/nei/js/scripts.js?sraguf"></script>
|
||
|
||
</body>
|
||
</html>
|