639 lines
31 KiB
Text
639 lines
31 KiB
Text
|
||
<!DOCTYPE html>
|
||
<html lang="en" dir="ltr">
|
||
<head>
|
||
<meta charset="utf-8" />
|
||
<meta name="description" content="Using a new experimental technique to fix faulty eye cells, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes one form of childhood blindness." />
|
||
<meta name="keywords" content="Leber Congenital Amaurosis,Gene Therapy,Rare Diseases" />
|
||
<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/new-gene-editing-technique-holds-potential-treating-childhood-blindness",
|
||
"name": "New gene-editing technique holds potential for treating childhood blindness",
|
||
"headline": "New gene-editing technique holds potential for treating childhood blindness",
|
||
"image": {
|
||
"@type": "ImageObject",
|
||
"representativeOfPage": "False",
|
||
"url": "https://www.nei.nih.gov/sites/default/files/styles/medium/public/2023-10/Nanoparticle.jpg?itok=egcGLBJV",
|
||
"width": "464",
|
||
"height": "450"
|
||
},
|
||
"datePublished": "2023-10-05T09:20:24-0400",
|
||
"dateModified": "2023-10-05T09:46:10-0400",
|
||
"author": {
|
||
"@type": "GovernmentOrganization",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/new-gene-editing-technique-holds-potential-treating-childhood-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/new-gene-editing-technique-holds-potential-treating-childhood-blindness",
|
||
"name": "National Eye Institute",
|
||
"url": "https://www.nei.nih.gov"
|
||
}
|
||
},
|
||
{
|
||
"@type": "WebPage",
|
||
"@id": "https://www.nei.nih.gov/about/news-and-events/news/new-gene-editing-technique-holds-potential-treating-childhood-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": "New gene-editing technique holds potential for treating childhood blindness",
|
||
"item": "https://www.nei.nih.gov/about/news-and-events/news/new-gene-editing-technique-holds-potential-treating-childhood-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="Oct 5, 2023, 9:46:10 AM" />
|
||
<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/new-gene-editing-technique-holds-potential-treating-childhood-blindness" />
|
||
<link rel="canonical" href="https://www.nei.nih.gov/about/news-and-events/news/new-gene-editing-technique-holds-potential-treating-childhood-blindness" />
|
||
<link rel="shortlink" href="https://www.nei.nih.gov/node/4044" />
|
||
|
||
<title>New gene-editing technique holds potential for treating childhood 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\/4044","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__4044 path-about-news-and-events-news-new-gene-editing-technique-holds-potential-treating-childhood-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/new-gene-editing-technique-holds-potential-treating-childhood-blindness" class="language-link is-active" lang="en" data-drupal-link-system-path="node/4044" aria-current="page">English</a>
|
||
</li>
|
||
<li>
|
||
<a href="/espanol/node/4044" class="language-link is-active" lang="es" data-drupal-link-system-path="node/4044" 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/new-gene-editing-technique-holds-potential-treating-childhood-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-vj6rxisgox3l8darfdswlzfwzc5e3de7xivl0nsp8n8" type="hidden" name="form_build_id" value="form-VJ6RxiSGOx3L8daRfDSwLZFwzC5e3de7XiVl0Nsp8n8" />
|
||
<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>
|
||
New gene-editing technique holds potential for treating childhood blindness
|
||
</li>
|
||
</ol>
|
||
</nav>
|
||
|
||
|
||
|
||
</div>
|
||
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<div class="r-page-header">
|
||
<div class="o-page-header">
|
||
|
||
<div id="block-nei-page-title">
|
||
|
||
|
||
|
||
|
||
|
||
|
||
<h1>
|
||
<span>New gene-editing technique holds potential for treating childhood 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="4044">
|
||
|
||
|
||
|
||
<div>
|
||
|
||
|
||
|
||
|
||
<div class="c-item__subtitle">
|
||
Using a new experimental technique to fix faulty eye cells, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes one form of childhood blindness.
|
||
</div>
|
||
|
||
|
||
<div class="c-item__date">
|
||
October 5, 2023
|
||
</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=55" hreflang="en">Leber Congenital Amaurosis</a>
|
||
<a href="/about/news-and-events/news?topic=56" hreflang="en">Rare Diseases</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-text">
|
||
|
||
|
||
|
||
|
||
<p>Using a new experimental technique to fix faulty eye cells, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes one form of childhood blindness. The scientists showed that their approach worked in lab-grown cells derived from a patient with the currently untreatable inherited disease Leber congenital amaurosis (LCA) and a mouse model that mimics the disease.</p>
|
||
|
||
<p>The method hinges on delivering gene-editing tool CRISPR base editor to retinal cells using silica nanocapsules. The proof-of-concept study restored the function of a protein that controls the flow of potassium ions in retinal tissue, which allows light-detecting cells to work properly.</p>
|
||
|
||
<p>“Our goal is to design a package that will carry CRISPR base editors to the retina,” said Bikash Pattnaik, co-investigator and associate professor of pediatrics at the UW School of Medicine and Public Health. “It will be able to introduce nanoparticles in the eye, and those nanoparticles will be designed to target the cell types identified for therapy.”</p>
|
||
|
||
<p>Pattnaik, affiliated with the Department of Ophthalmology and Visual Sciences, led a team including scientists and engineers from UW–Madison, Harvard, and MIT that published the study in the Journal of Clinical Investigation. The researchers are seeking to develop treatments for two forms of childhood blindness caused by retinal gene mutations, LCA and Best disease, which is also known as Best vitelliform macular dystrophy. LCA begins in infancy and causes extreme farsightedness, sensitivity to light, and involuntary eye movements. Best disease, usually diagnosed in childhood, causes macular degeneration and loss of central vision, visual acuity, and color perception.</p>
|
||
|
||
<p>The team reasoned that silica nanocapsules do not interact with the body, lowering the risk of problematic and unpredictable immune system responses that viral methods may elicit. Another advantage of the technique is its specificity. “The rapid pace of genetic diagnosis development has enabled accuracy in identifying disease-causing mutations, and we used base editing to correct a specific error in the DNA sequence of patients, overcoming several challenges through the collaborative team science approach,” said research associate Meha Kabra.</p>
|
||
|
||
|
||
</div>
|
||
|
||
|
||
|
||
<div class="c-link">
|
||
|
||
<a href="https://www.med.wisc.edu/news-and-events/2023/september/gene-editing-technique-childhood-blindness/" class="c-link__link">U. Wisconsin news story</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-fafb48cb1666409862adb12181aeee5423de16cb239be04607bb3efcc2fecd24 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>
|