CINXE.COM
Vectors 101 | ASGCT - American Society of Gene & Cell Therapy |
<!DOCTYPE html> <html xmlns="http://www.w3.org/1999/xhtml"> <head> <!-- Google Tag Manager --> <script type="text/javascript">(function (w, d, s, l, i) { w[l] = w[l] || []; w[l].push({ 'gtm.start': new Date().getTime(), event: 'gtm.js' }); var f = d.getElementsByTagName(s)[0], j = d.createElement(s), dl = l != 'dataLayer' ? '&l=' + l : ''; j.async = true; j.src = 'https://www.googletagmanager.com/gtm.js?id=' + i + dl; f.parentNode.insertBefore(j, f); })(window, document, 'script', 'dataLayer', 'GTM-NFW5DS9');</script> <!-- End Google Tag Manager --> <!-- Begin Informz Tracking --> <script type="text/javascript"> //------------------------------------------------------- var z_account = "2632C1D8-6432-4D3A-93D6-08AAECC53F86"; var z_collector = "asgct.informz.net"; var z_cookieDomain = ".asgct.org"; //------------------------------------------------------- (function (e, o, n, r, t, a, s) { e[t] || (e.GlobalSnowplowNamespace = e.GlobalSnowplowNamespace || [], e.GlobalSnowplowNamespace.push(t), e[t] = function () { (e[t].q = e[t].q || []).push(arguments) }, e[t].q = e[t].q || [], a = o.createElement(n), s = o.getElementsByTagName(n)[0], a.async = 1, a.src = r, s.parentNode.insertBefore(a, s)) }(window, document, "script", "https://cdn.informz.net/web_trk/sp.js", "informz_trk")), informz_trk("newTracker", "infz", z_collector + "/web_trk/collector/", { appId: z_account, cookieDomain: z_cookieDomain }), informz_trk("setUserIdFromLocation", "_zs"), informz_trk("enableActivityTracking", 30, 15); informz_trk("trackPageView", null); </script> <!-- End Informz Tracking --> <meta charset="utf-8" /> <meta http-equiv="X-UA-Compatible" content="IE=edge" /> <meta name="viewport" content="width=device-width,user-scalable=no,maximum-scale=1.0,initial-scale=1.0" /> <link href="/BlueKey/pe_assets/build/styles.css" rel="stylesheet" /> <script type="text/javascript" defer="defer" src="https://extend.vimeocdn.com/ga/127349852.js"></script> <meta name="twitter:card" content="summary_large_image" /> <meta name="twitter:site" content="https://twitter.com/ASGCTherapy" <meta name="twitter:creator" content="@ASGCTherapy" <meta name="twitter:title" content="Vectors 101" <meta name="twitter:description" content="Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell." <meta name="twitter:image" content="~/attachment.aspx" <meta name="description" content="Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell." /> <meta charset="UTF-8" /> <link href="/App_Themes/Default/Images/favicon.ico" type="image/x-icon" rel="shortcut icon" /> <link href="/App_Themes/Default/Images/favicon.ico" type="image/x-icon" rel="icon" /> <title> Vectors 101 | ASGCT - American Society of Gene & Cell Therapy | </title></head> <body class="LTR IE IE7 ENUS ContentBody"> <!-- Google Tag Manager (noscript) --> <noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-NFW5DS9" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript> <!-- End Google Tag Manager (noscript) --> <form method="post" action="/gene-therapy-101/vectors-101" id="aspnetForm"> <div class="aspNetHidden"> <input type="hidden" name="__CMSCsrfToken" id="__CMSCsrfToken" value="umsvKMRt6j9eA3eERk2dWMmdJuDYA4ebq2E1xRmG1XPAGX9TnUBFaCfCcp+ZYF535/MwxlB5AwudA978mDpAjEFefYM=" /> <input type="hidden" name="__EVENTTARGET" id="__EVENTTARGET" value="" /> <input type="hidden" name="__EVENTARGUMENT" id="__EVENTARGUMENT" value="" /> <input type="hidden" name="__VIEWSTATE" id="__VIEWSTATE" value="/wEPDwUKMjEyMjY0MDQ1Ng8WAh4TVmFsaWRhdGVSZXF1ZXN0TW9kZQIBFgJmD2QWBGYPZBYCZg9kFgICAQ9kFgICAQ8WAh4EVGV4dAWRAzxtZXRhIG5hbWU9InR3aXR0ZXI6c2l0ZSIgY29udGVudD0iaHR0cHM6Ly90d2l0dGVyLmNvbS9BU0dDVGhlcmFweSINCjxtZXRhIG5hbWU9InR3aXR0ZXI6Y3JlYXRvciIgY29udGVudD0iQEFTR0NUaGVyYXB5Ig0KPG1ldGEgbmFtZT0idHdpdHRlcjp0aXRsZSIgY29udGVudD0iVmVjdG9ycyAxMDEiDQo8bWV0YSBuYW1lPSJ0d2l0dGVyOmRlc2NyaXB0aW9uIiBjb250ZW50PSJWZWN0b3JzIGFyZSBlc3NlbnRpYWxseSB2ZWhpY2xlcyBkZXNpZ25lZCB0byBkZWxpdmVyIHRoZXJhcGV1dGljIGdlbmV0aWMgbWF0ZXJpYWwsIHN1Y2ggYXMgYSB3b3JraW5nIGdlbmUsIGRpcmVjdGx5IGludG8gYSBjZWxsLiINCjxtZXRhIG5hbWU9InR3aXR0ZXI6aW1hZ2UiIGNvbnRlbnQ9In4vYXR0YWNobWVudC5hc3B4Ig0KZAIBDxYCHgZhY3Rpb24FHS9nZW5lLXRoZXJhcHktMTAxL3ZlY3RvcnMtMTAxFgQCAw9kFghmD2QWBmYPDxYCHgdWaXNpYmxlZ2QWAgIBDxYCHwNnFgQCAQ8WAh8BBUxodHRwczovL3BhdGllbnRlZHVjYXRpb24uYXNnY3Qub3JnL25ld3NsZXR0ZXIvc3Vic2NyaWJlLXRvLXRoZS1wYXRpZW50LXByZXNzZAIDDxYCHwEFSlN1YnNjcmliZSB0b2RheSB0byBBU0dDVCdzIHBhdGllbnQtY2VudGVyZWQgbmV3c2xldHRlciwgVGhlIFBhdGllbnQgUHJlc3MhZAIBDxYCHgtfIUl0ZW1Db3VudAIIFhACAQ9kFgRmDxUCES9nZW5lLXRoZXJhcHktMTAxF0dlbmUgJiBDZWxsIFRoZXJhcHkgMTAxZAIBDxYCHwQCCBYQAgEPZBYEZg8VASUvZ2VuZS10aGVyYXB5LTEwMS9nZW5lLXRoZXJhcHktYmFzaWNzZAICDxUBE0dlbmUgVGhlcmFweSBCYXNpY3NkAgIPZBYEZg8VASUvZ2VuZS10aGVyYXB5LTEwMS9jZWxsLXRoZXJhcHktYmFzaWNzZAICDxUBE0NlbGwgVGhlcmFweSBCYXNpY3NkAgMPZBYEZg8VASkvZ2VuZS10aGVyYXB5LTEwMS9nZW5lLXRoZXJhcHktYXBwcm9hY2hlc2QCAg8VARdHZW5lIFRoZXJhcHkgQXBwcm9hY2hlc2QCBA9kFgRmDxUBHi9nZW5lLXRoZXJhcHktMTAxL2dlbmUtZWRpdGluZ2QCAg8VAQxHZW5lIEVkaXRpbmdkAgUPZBYEZg8VASEvZ2VuZS10aGVyYXB5LTEwMS9jbGluaWNhbC10cmlhbHNkAgIPFQEPQ2xpbmljYWwgVHJpYWxzZAIGD2QWBGYPFQEdL2dlbmUtdGhlcmFweS0xMDEvdmVjdG9ycy0xMDFkAgIPFQELVmVjdG9ycyAxMDFkAgcPZBYEZg8VARovZ2VuZS10aGVyYXB5LTEwMS9nbG9zc2FyeWQCAg8VAQhHbG9zc2FyeWQCCA9kFgRmDxUBIi9nZW5lLXRoZXJhcHktMTAxL2NvdmlkMTktdmFjY2luZXNkAgIPFQEIQ09WSUQtMTlkAgIPZBYEZg8VAhMvZGlzZWFzZS10cmVhdG1lbnRzFENvbmRpdGlvbiBUcmVhdG1lbnRzZAIBDxYCHwQCFBYoAgEPZBYEZg8VATsvZGlzZWFzZS10cmVhdG1lbnRzL2ZkYS1hcHByb3ZlZC1jZWxsLWdlbmUtdGhlcmFweS1wcm9kdWN0c2QCAg8VASlGREEtQXBwcm92ZWQgQ2VsbCAmIEdlbmUgVGhlcmFweSBQcm9kdWN0c2QCAg9kFgRmDxUBGi9kaXNlYXNlLXRyZWF0bWVudHMvYmF0dGVuZAICDxUBDkJhdHRlbiBEaXNlYXNlZAIDD2QWBGYPFQEjL2Rpc2Vhc2UtdHJlYXRtZW50cy9ibG9vZC1kaXNvcmRlcnNkAgIPFQEoQmV0YSBUaGFsYXNzZW1pYSAmIE90aGVyIEJsb29kIERpc29yZGVyc2QCBA9kFgRmDxUBIi9kaXNlYXNlLXRyZWF0bWVudHMvbGV1a29keXN0cm9waHlkAgIPFQEMQ0FMRCBhbmQgTUxEZAIFD2QWBGYPFQEgL2Rpc2Vhc2UtdHJlYXRtZW50cy9jYXItdC1iYXNpY3NkAgIPFQEMQ0FSLVQgQmFzaWNzZAIGD2QWBGYPFQEZL2Rpc2Vhc2UtdHJlYXRtZW50cy9mYWJyeWQCAg8VAQ1GYWJyeSBEaXNlYXNlZAIHD2QWBGYPFQEjL2Rpc2Vhc2UtdHJlYXRtZW50cy9nYXVjaGVyLWRpc2Vhc2VkAgIPFQEPR2F1Y2hlciBEaXNlYXNlZAIID2QWBGYPFQEmL2Rpc2Vhc2UtdHJlYXRtZW50cy9nbTEtZ2FuZ2xpb3NpZG9zaXNkAgIPFQESR00xIEdhbmdsaW9zaWRvc2lzZAIJD2QWBGYPFQEqL2Rpc2Vhc2UtdHJlYXRtZW50cy9nbTItdGF5LXNhY2hzLXNhbmRob2ZmZAICDxUBHlRheS1TYWNocyBhbmQgU2FuZGhvZmYgRGlzZWFzZWQCCg9kFgRmDxUBHi9kaXNlYXNlLXRyZWF0bWVudHMvaGVtb3BoaWxpYWQCAg8VAQpIZW1vcGhpbGlhZAILD2QWBGYPFQEnL2Rpc2Vhc2UtdHJlYXRtZW50cy9odW50aW5ndG9ucy1kaXNlYXNlZAICDxUBFEh1bnRpbmd0b24ncyBEaXNlYXNlZAIMD2QWBGYPFQEuL2Rpc2Vhc2UtdHJlYXRtZW50cy9pbmhlcml0ZWQtcmV0aW5hbC1kaXNlYXNlc2QCAg8VARpJbmhlcml0ZWQgUmV0aW5hbCBEaXNlYXNlc2QCDQ9kFgRmDxUBFy9kaXNlYXNlLXRyZWF0bWVudHMvbXBzZAICDxUBG011Y29wb2x5c2FjY2hhcmlkb3NlcyAoTVBTKWQCDg9kFgRmDxUBJi9kaXNlYXNlLXRyZWF0bWVudHMvbXVzY3VsYXItZHlzdHJvcGh5ZAICDxUBEk11c2N1bGFyIER5c3Ryb3BoeWQCDw9kFgRmDxUBIS9kaXNlYXNlLXRyZWF0bWVudHMvcG9tcGUtZGlzZWFzZWQCAg8VAQ1Qb21wZSBEaXNlYXNlZAIQD2QWBGYPFQEmL2Rpc2Vhc2UtdHJlYXRtZW50cy9wYXJraW5zb25zLWRpc2Vhc2VkAgIPFQETUGFya2luc29uJ3MgRGlzZWFzZWQCEQ9kFgRmDxUBJy9kaXNlYXNlLXRyZWF0bWVudHMvc2lja2xlLWNlbGwtZGlzZWFzZWQCAg8VARNTaWNrbGUgQ2VsbCBEaXNlYXNlZAISD2QWBGYPFQEXL2Rpc2Vhc2UtdHJlYXRtZW50cy9zbWFkAgIPFQEXU3BpbmFsIE11c2N1bGFyIEF0cm9waHlkAhMPZBYEZg8VASIvZGlzZWFzZS10cmVhdG1lbnRzL3dpbHNvbi1kaXNlYXNlZAICDxUBDldpbHNvbiBEaXNlYXNlZAIUD2QWBGYPFQEZL2Rpc2Vhc2UtdHJlYXRtZW50cy94bG10bWQCAg8VARxYLUxpbmtlZCBNeW90dWJ1bGFyIE15b3BhdGh5ZAIDD2QWBGYPFQIQL3BhdGllbnQtam91cm5leQ9QYXRpZW50IEpvdXJuZXlkAgEPFgIfBAIKFhQCAQ9kFgRmDxUBLi9wYXRpZW50LWpvdXJuZXkvaWRlbnRpZnlpbmctYS1nZW5ldGljLWRpc2Vhc2VkAgIPFQEdSWRlbnRpZnlpbmcgYSBHZW5ldGljIERpc2Vhc2VkAgIPZBYEZg8VASwvcGF0aWVudC1qb3VybmV5L25leHQtc3RlcHMtd2l0aC1hLWRpYWdub3Npc2QCAg8VARtOZXh0IFN0ZXBzIFdpdGggYSBEaWFnbm9zaXNkAgMPZBYEZg8VATEvcGF0aWVudC1qb3VybmV5L3BhdGllbnQtZm9jdXNlZC1wb2xpY3ktcG9zaXRpb25zZAICDxUBIFBhdGllbnQtRm9jdXNlZCBQb2xpY3kgUG9zaXRpb25zZAIED2QWBGYPFQElL3BhdGllbnQtam91cm5leS9zdGltdWxhdGluZy1yZXNlYXJjaGQCAg8VARRTdGltdWxhdGluZyBSZXNlYXJjaGQCBQ9kFgRmDxUBLS9wYXRpZW50LWpvdXJuZXkvY29uc2lkZXJpbmctYS1jbGluaWNhbC10cmlhbGQCAg8VARxDb25zaWRlcmluZyBhIENsaW5pY2FsIFRyaWFsZAIGD2QWBGYPFQE1L3BhdGllbnQtam91cm5leS9ldGhpY2FsLWlzc3Vlcy1nZXJtbGluZS1nZW5lLWVkaXRpbmdkAgIPFQElRXRoaWNhbCBJc3N1ZXM6IEdlcm1saW5lIEdlbmUgRWRpdGluZ2QCBw9kFgRmDxUBPC9wYXRpZW50LWpvdXJuZXkvZXRoaWNhbC1pc3N1ZXMtaWxsZWdpdGltYXRlLWNsaW5pY2FsLXRyaWFsc2QCAg8VAStFdGhpY2FsIElzc3VlczogVW5wcm92ZW4gU3RlbSBDZWxsIFByb2R1Y3RzZAIID2QWBGYPFQElL3BhdGllbnQtam91cm5leS9nZW5lLXRoZXJhcHktY2VudGVyc2QCAg8VARRHZW5lIFRoZXJhcHkgQ2VudGVyc2QCCQ9kFgRmDxUBJy9wYXRpZW50LWpvdXJuZXkvY2xpbmljYWwtdHJpYWxzLWZpbmRlcmQCAg8VARZDbGluaWNhbCBUcmlhbHMgRmluZGVyZAIKD2QWBGYPFQEaL3BhdGllbnQtam91cm5leS9vbi1kZW1hbmRkAgIPFQEST24gRGVtYW5kIFdlYmluYXJzZAIED2QWBGYPFQILL25ld3NsZXR0ZXIKTmV3c2xldHRlcmQCAQ8WAh8EAgEWAgIBD2QWBGYPFQEqL25ld3NsZXR0ZXIvc3Vic2NyaWJlLXRvLXRoZS1wYXRpZW50LXByZXNzZAICDxUBHlN1YnNjcmliZSB0byBUaGUgUGF0aWVudCBQcmVzc2QCBQ9kFgRmDxUCDC9xdWljay10YWtlcwtRdWljayBUYWtlc2QCAQ8WAh8EAgUWCgIBD2QWBGYPFQE6L3F1aWNrLXRha2VzL2ZvY3VzZWQtdWx0cmFzb3VuZC1pbi1jZWxsLWFuZC1nZW5lLXRoZXJhcGllc2QCAg8VARpGb2N1c2VkIFVsdHJhc291bmQgaW4gQ0dUc2QCAg9kFgRmDxUBNy9xdWljay10YWtlcy9hY2Nlc3MtdG8tYXBwcm92ZWQtY2VsbC1hbmQtZ2VuZS10aGVyYXBpZXNkAgIPFQEXQWNjZXNzIHRvIEFwcHJvdmVkIENHVHNkAgMPZBYEZg8VAR0vcXVpY2stdGFrZXMvaW5mb3JtZWQtY29uc2VudGQCAg8VARBJbmZvcm1lZCBDb25zZW50ZAIED2QWBGYPFQE/L3F1aWNrLXRha2VzL3N0YXRlLW9mLXRoZS1maWVsZC1jZ3QtaGlnaGxpZ2h0cy1hbmQtYXBwcm92YWxzLTIwZAICDxUBOFN0YXRlIG9mIHRoZSBGaWVsZDogQ0dUIEhpZ2hsaWdodHMgYW5kIEFwcHJvdmFscyBvZiAyMDIzZAIFD2QWBGYPFQE7L3F1aWNrLXRha2VzL2dlbmV0aWMtdGVzdGluZy1hbmQtY2xpbmljYWwtdHJpYWwtZWxpZ2liaWxpdHlkAgIPFQEuR2VuZXRpYyBUZXN0aW5nIGFuZCBDbGluaWNhbCBUcmlhbCBFbGlnaWJpbGl0eWQCBg9kFgRmDxUCBy9ldmVudHMGRXZlbnRzZAIBDxYCHwQCAhYEAgEPZBYEZg8VATovZXZlbnRzL2VtcG93ZXJpbmctcGF0aWVudHMtMjAyNS1hLWNlbGwtYW5kLWdlbmUtdGhlcmFwaWVzZAICDxUBOkVtcG93ZXJpbmcgUGF0aWVudHMgMjAyNTogQSBDZWxsIGFuZCBHZW5lIFRoZXJhcGllcyBTdW1taXRkAgIPZBYEZg8VATEvZXZlbnRzL2FubnVhbC1tZWV0aW5nLXBhdGllbnQtYWR2b2NhdGUtb2ZmZXJpbmdzZAICDxUBKUFubnVhbCBNZWV0aW5nLVBhdGllbnQgQWR2b2NhdGUgT2ZmZXJpbmdzZAIHD2QWBGYPFQIML2Fib3V0LWFzZ2N0BUFib3V0ZAIBDxYCHwQCBBYIAgEPZBYEZg8VARUvYWJvdXQtYXNnY3Qvb3ZlcnZpZXdkAgIPFQEIT3ZlcnZpZXdkAgIPZBYEZg8VARgvYWJvdXQtYXNnY3QvYWJvdXQtYXNnY3RkAgIPFQELQWJvdXQgQVNHQ1RkAgMPZBYEZg8VAREvYWJvdXQtYXNnY3QvbmV3c2QCAg8VAQpBU0dDVCBOZXdzZAIED2QWBGYPFQEbL2Fib3V0LWFzZ2N0L3ByaXZhY3ktcG9saWN5ZAICDxUBDlByaXZhY3kgUG9saWN5ZAIID2QWBGYPFQIIL3N1cHBvcnQHU3VwcG9ydGQCAQ8WAh8EAv////8PZAICDxYCHwNoZAIBDw8WCh4IU2l0ZU5hbWUFEFBhdGllbnRFZHVjYXRpb24eDENhY2hlTWludXRlcwIKHgtEZWZhdWx0UGF0aAUdL0dlbmUtVGhlcmFweS0xMDEvVmVjdG9ycy0xMDEeBFBhdGgFHS9HZW5lLVRoZXJhcHktMTAxL1ZlY3RvcnMtMTAxHhRTZWxlY3ROb2Rlc1N0YXJ0UGF0aAUBL2RkAgIPFgIfBAIBFgJmD2QWBGYPFQGsATxkaXYgY2xhc3M9ImMtbGVhZGVyLWNvbnRlbnQiPjxwPlZlY3RvcnMgYXJlIGVzc2VudGlhbGx5IHZlaGljbGVzIGRlc2lnbmVkIHRvIGRlbGl2ZXIgdGhlcmFwZXV0aWMgZ2VuZXRpYyBtYXRlcmlhbCwgc3VjaCBhcyBhIHdvcmtpbmcgZ2VuZSwgZGlyZWN0bHkgaW50byBhIGNlbGwuIDwvcD48L2Rpdj5kAgEPZBYCZg8VAwAAAGQCAw8UKwACDxYEHgtfIURhdGFCb3VuZGcfBAIJZGQWAmYPZBYUAgEPFgIfAQUNUmVsYXRlZCBQYWdlc2QCAw9kFgRmDxUDGGNsYXNzPSJjLW5hdi1sb2NhbC1pdGVtIiUvZ2VuZS10aGVyYXB5LTEwMS9nZW5lLXRoZXJhcHktYmFzaWNzE0dlbmUgVGhlcmFweSBCYXNpY3NkAgEPFgIfBAL/////D2QCBA9kFgRmDxUDGGNsYXNzPSJjLW5hdi1sb2NhbC1pdGVtIiUvZ2VuZS10aGVyYXB5LTEwMS9jZWxsLXRoZXJhcHktYmFzaWNzE0NlbGwgVGhlcmFweSBCYXNpY3NkAgEPFgIfBAL/////D2QCBQ9kFgRmDxUDGGNsYXNzPSJjLW5hdi1sb2NhbC1pdGVtIikvZ2VuZS10aGVyYXB5LTEwMS9nZW5lLXRoZXJhcHktYXBwcm9hY2hlcxdHZW5lIFRoZXJhcHkgQXBwcm9hY2hlc2QCAQ8WAh8EAv////8PZAIGD2QWBGYPFQMYY2xhc3M9ImMtbmF2LWxvY2FsLWl0ZW0iHi9nZW5lLXRoZXJhcHktMTAxL2dlbmUtZWRpdGluZwxHZW5lIEVkaXRpbmdkAgEPFgIfBAL/////D2QCBw9kFgRmDxUDGGNsYXNzPSJjLW5hdi1sb2NhbC1pdGVtIiEvZ2VuZS10aGVyYXB5LTEwMS9jbGluaWNhbC10cmlhbHMPQ2xpbmljYWwgVHJpYWxzZAIBDxYCHwQC/////w9kAggPZBYEZg8VAyJjbGFzcz0iYy1uYXYtbG9jYWwtaXRlbSBpcy1hY3RpdmUiHS9nZW5lLXRoZXJhcHktMTAxL3ZlY3RvcnMtMTAxC1ZlY3RvcnMgMTAxZAIBDxYCHwQC/////w9kAgkPZBYEZg8VAxhjbGFzcz0iYy1uYXYtbG9jYWwtaXRlbSIiL2dlbmUtdGhlcmFweS0xMDEvY292aWQxOS12YWNjaW5lcwhDT1ZJRC0xOWQCAQ8WAh8EAv////8PZAIKD2QWBGYPFQMYY2xhc3M9ImMtbmF2LWxvY2FsLWl0ZW0iMi9nZW5lLXRoZXJhcHktMTAxL2x1bmNoLWxlYXJuLWdlbmUtdGhlcmFweS1mb3Itc21hI0x1bmNoICYgTGVhcm46IEdlbmUgVGhlcmFweSBmb3IgU01BZAIBDxYCHwQC/////w9kAgsPZBYEZg8VAxhjbGFzcz0iYy1uYXYtbG9jYWwtaXRlbSIiL2dlbmUtdGhlcmFweS0xMDEvaGVscGZ1bC13ZWJzaXRlcxBIZWxwZnVsIFdlYnNpdGVzZAIBDxYCHwQC/////w9kAgUPZBYEAgEPFgIfBAIDFgZmD2QWAmYPFQEXY29sdW1uIGMtZm9vdGVyLXNlY3Rpb25kAgEPZBYCZg8VASVjb2x1bW4gaXMtdHdvLWZpZnRocyBjLWZvb3Rlci1zZWN0aW9uZAICD2QWBGYPFQEXY29sdW1uIGMtZm9vdGVyLXNlY3Rpb25kAgEPZBYCAgEPZBYCZg9kFgJmD2QWCgIBD2QWAmYPFQMfaHR0cHM6Ly90d2l0dGVyLmNvbS9BU0dDVGhlcmFweQd0d2l0dGVyB1R3aXR0ZXJkAgIPZBYCZg8VAyRodHRwczovL3d3dy5mYWNlYm9vay5jb20vQVNHQ1RoZXJhcHkIZmFjZWJvb2sIRmFjZWJvb2tkAgMPZBYCZg8VAyVodHRwczovL3d3dy5pbnN0YWdyYW0uY29tL2FzZ2N0aGVyYXB5CWluc3RhZ3JhbQlJbnN0YWdyYW1kAgQPZBYCZg8VAyZodHRwczovL3d3dy5saW5rZWRpbi5jb20vY29tcGFueS9hc2djdAhsaW5rZWRpbghMaW5rZWRJbmQCBQ9kFgJmDxUDImh0dHBzOi8vd3d3LnlvdXR1YmUuY29tL3VzZXIvQVNHQ1QHeW91dHViZQdZb3VUdWJlZAIDDxYCHwEFbDxwIGNsYXNzPSJpcy1zbWFsbCI+JmNvcHk7IDIwMjQmbmJzcDtBbGwgcmlnaHRzIHJlc2VydmVkLiBBbWVyaWNhbiBTb2NpZXR5IG9mIEdlbmUgJmFtcDsgQ2VsbCBUaGVyYXB5LjwvcD4NCmQYAQUWY3RsMDAkcGxjTWFpbiRsdlN1Yk5hdg8UKwAOZGRkZGRkZDwrAAkAAglkZGRmAv////8PZP+pf6DdZ8lQDiM4mcPs2L+1nQ2n" /> </div> <script type="text/javascript"> //<![CDATA[ var theForm = document.forms['aspnetForm']; if (!theForm) { theForm = document.aspnetForm; } function __doPostBack(eventTarget, eventArgument) { if (!theForm.onsubmit || (theForm.onsubmit() != false)) { theForm.__EVENTTARGET.value = eventTarget; theForm.__EVENTARGUMENT.value = eventArgument; theForm.submit(); } } //]]> </script> <script src="/WebResource.axd?d=UZQNzmZXG1IdUaHxtavOjRvGEvOmz1kvw97PqEaxzBy7Ys-tUotw-RqgdbsvC3g_7cXjehqAdA_N6kbuK73WBAVGdxo1&t=638562384440000000" type="text/javascript"></script> <input type="hidden" name="lng" id="lng" value="en-US" /> <script type="text/javascript"> //<![CDATA[ function PM_Postback(param) { if (window.top.HideScreenLockWarningAndSync) { window.top.HideScreenLockWarningAndSync(1080); } if(window.CMSContentManager) { CMSContentManager.allowSubmit = true; }; __doPostBack('ctl00$CMSPortalManager1$am',param); } function PM_Callback(param, callback, ctx) { if (window.top.HideScreenLockWarningAndSync) { window.top.HideScreenLockWarningAndSync(1080); }if (window.CMSContentManager) { CMSContentManager.storeContentChangedStatus(); };WebForm_DoCallback('ctl00$CMSPortalManager1$am',param,callback,ctx,null,true); } //]]> </script> <script src="/ScriptResource.axd?d=WTJtoz6EJe-loUBJ9stbRxqzgpVp8oo0KdZGkhGfcu6Lr7kb5LeNfx21FKi8mxEL1oDZOLAxiAqERpClgBx-A6lZ0DJ1qKcDZB1NuIbRd8o9S7aG0&t=7c776dc1" type="text/javascript"></script> <script src="/ScriptResource.axd?d=y6KGkkRaceYF_mkApXCXMQ_rMQVX59BlJ-ut-lQDHw88bR5bbS86epdVNwimj01duE6_OophLX4sBTOgb8_5dNLaY2Mx5DHMJj7Rbr4kPMV1-AOS5VF5-QEC8oG-_-aHoWoyqA2&t=7c776dc1" type="text/javascript"></script> <script type="text/javascript"> //<![CDATA[ var CMS = CMS || {}; CMS.Application = { "isDialog": false, "isDebuggingEnabled": false, "applicationUrl": "/", "isRTL": "false", "imagesUrl": "/CMSPages/GetResource.ashx?image=%5bImages.zip%5d%2f" }; //]]> </script> <div class="aspNetHidden"> <input type="hidden" name="__VIEWSTATEGENERATOR" id="__VIEWSTATEGENERATOR" value="AD1448C3" /> <input type="hidden" name="__EVENTVALIDATION" id="__EVENTVALIDATION" value="/wEdAAMjHpt1eiQ7DW6pFfQZS12MDMN6QAizBz4KoPbUd79oAZD1NQdfl/cWuar5Vh7dGPS984wj/hO5KfetjtOCX3d37sytfA==" /> </div> <script type="text/javascript"> //<![CDATA[ Sys.WebForms.PageRequestManager._initialize('ctl00$manScript', 'aspnetForm', ['tctl00$ctxM',''], [], [], 90, 'ctl00'); //]]> </script> <div id="ctl00_ctxM"> </div> <header class="c-header"> <a href="#content" class="show-on-focus">Skip to main content</a> <div class="c-header-utilities"> <div class="container"> <div class="columns is-vcentered"> <div class="column"> <a href="https://asgct.org" class="is-external">ASGCT.org</a> </div> <div class="column is-half"> <a href="/"> <div class="c-header-images"> <div class="c-header-image"> <img src="/PatientEducation/media/PatientEducation/ASGCT_Web_Horz_Navy.png?ext=.png" alt="" /> </div> <div class="c-header-image"> <img src="/PatientEducation/media/PatientEducation/temp-pe-logo.png?ext=.png" alt="Patient Education Logo" /> </div> </div> </a> </div> <!--/search-results?searchtext=<enteredtext>--> <div class="column"> <div class="c-header-search"> <div class="c-header-search-wrap"> <input name="keyword" type="text" id="searchtext" placeholder="Search" aria-required="false" /> <label for="searchtext" class="is-sr-only">Keyword search</label> <button type="button" data-encode="searchtext" data-path="/search-results" data-query="searchtext"> <span class="is-sr-only">Search</span> <span class="c-search-icon"></span> </button> </div> </div> </div> </div> </div> </div> <div class="c-header-utilities-mobile"> <div class="c-header-images"> <div class="c-header-image"> <a href="https://asgct.org" class="is-external"><img src="/PatientEducation/media/PatientEducation/ASGCT_Web_Horz_Navy.png?ext=.png" alt="" /></a> </div> <div class="c-header-image"> <a href="/"><img src="/PatientEducation/media/PatientEducation/temp-pe-logo.png?ext=.png" alt="Patient Education Logo" /></a> </div> </div> <button class="c-nav-toggle" type="button" aria-controls="c-nav-main" aria-expanded="false" data-togglevisibility="data-togglevisibility"> <p class="is-sr-only">Open Navigation</p> <p class="is-sr-only" aria-hidden="true">Close Navigation</p> <span class="c-nav-toggle-menu"></span> </button> </div> <div id="ctl00_plcMain_bkSiteHeader_headerBanner"> <div class="header-alert"> <div class="container"> <p id="ctl00_plcMain_bkSiteHeader_pBanner"><b><a href="https://patienteducation.asgct.org/newsletter/subscribe-to-the-patient-press">Subscribe today to ASGCT's patient-centered newsletter, The Patient Press!</a></b></p> </div> </div> </div> <nav class="c-nav-main" id="c-nav-main" aria-hidden="true"> <div class="container"> <div class="c-header-search is-hidden-desktop"> <div class="c-header-search-wrap"> <input name="" type="text" id="global-search" placeholder="Search" aria-required="false" /> <label for="global-search" class="is-sr-only">Keyword search</label> <button type="button" data-encode="global-search" data-path="/search-results" data-query="searchtext"> <span class="is-sr-only">Search</span> <span class="c-search-icon"></span> </button> </div> </div> <ul> <li> <a href="/gene-therapy-101">Gene & Cell Therapy 101</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/gene-therapy-101/gene-therapy-basics">Gene Therapy Basics</a> </li> <li> <a href="/gene-therapy-101/cell-therapy-basics">Cell Therapy Basics</a> </li> <li> <a href="/gene-therapy-101/gene-therapy-approaches">Gene Therapy Approaches</a> </li> <li> <a href="/gene-therapy-101/gene-editing">Gene Editing</a> </li> <li> <a href="/gene-therapy-101/clinical-trials">Clinical Trials</a> </li> <li> <a href="/gene-therapy-101/vectors-101">Vectors 101</a> </li> <li> <a href="/gene-therapy-101/glossary">Glossary</a> </li> <li> <a href="/gene-therapy-101/covid19-vaccines">COVID-19</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/disease-treatments">Condition Treatments</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/disease-treatments/fda-approved-cell-gene-therapy-products">FDA-Approved Cell & Gene Therapy Products</a> </li> <li> <a href="/disease-treatments/batten">Batten Disease</a> </li> <li> <a href="/disease-treatments/blood-disorders">Beta Thalassemia & Other Blood Disorders</a> </li> <li> <a href="/disease-treatments/leukodystrophy">CALD and MLD</a> </li> <li> <a href="/disease-treatments/car-t-basics">CAR-T Basics</a> </li> <li> <a href="/disease-treatments/fabry">Fabry Disease</a> </li> <li> <a href="/disease-treatments/gaucher-disease">Gaucher Disease</a> </li> <li> <a href="/disease-treatments/gm1-gangliosidosis">GM1 Gangliosidosis</a> </li> <li> <a href="/disease-treatments/gm2-tay-sachs-sandhoff">Tay-Sachs and Sandhoff Disease</a> </li> <li> <a href="/disease-treatments/hemophilia">Hemophilia</a> </li> <li> <a href="/disease-treatments/huntingtons-disease">Huntington's Disease</a> </li> <li> <a href="/disease-treatments/inherited-retinal-diseases">Inherited Retinal Diseases</a> </li> <li> <a href="/disease-treatments/mps">Mucopolysaccharidoses (MPS)</a> </li> <li> <a href="/disease-treatments/muscular-dystrophy">Muscular Dystrophy</a> </li> <li> <a href="/disease-treatments/pompe-disease">Pompe Disease</a> </li> <li> <a href="/disease-treatments/parkinsons-disease">Parkinson's Disease</a> </li> <li> <a href="/disease-treatments/sickle-cell-disease">Sickle Cell Disease</a> </li> <li> <a href="/disease-treatments/sma">Spinal Muscular Atrophy</a> </li> <li> <a href="/disease-treatments/wilson-disease">Wilson Disease</a> </li> <li> <a href="/disease-treatments/xlmtm">X-Linked Myotubular Myopathy</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/patient-journey">Patient Journey</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/patient-journey/identifying-a-genetic-disease">Identifying a Genetic Disease</a> </li> <li> <a href="/patient-journey/next-steps-with-a-diagnosis">Next Steps With a Diagnosis</a> </li> <li> <a href="/patient-journey/patient-focused-policy-positions">Patient-Focused Policy Positions</a> </li> <li> <a href="/patient-journey/stimulating-research">Stimulating Research</a> </li> <li> <a href="/patient-journey/considering-a-clinical-trial">Considering a Clinical Trial</a> </li> <li> <a href="/patient-journey/ethical-issues-germline-gene-editing">Ethical Issues: Germline Gene Editing</a> </li> <li> <a href="/patient-journey/ethical-issues-illegitimate-clinical-trials">Ethical Issues: Unproven Stem Cell Products</a> </li> <li> <a href="/patient-journey/gene-therapy-centers">Gene Therapy Centers</a> </li> <li> <a href="/patient-journey/clinical-trials-finder">Clinical Trials Finder</a> </li> <li> <a href="/patient-journey/on-demand">On Demand Webinars</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/newsletter">Newsletter</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/newsletter/subscribe-to-the-patient-press">Subscribe to The Patient Press</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/quick-takes">Quick Takes</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/quick-takes/focused-ultrasound-in-cell-and-gene-therapies">Focused Ultrasound in CGTs</a> </li> <li> <a href="/quick-takes/access-to-approved-cell-and-gene-therapies">Access to Approved CGTs</a> </li> <li> <a href="/quick-takes/informed-consent">Informed Consent</a> </li> <li> <a href="/quick-takes/state-of-the-field-cgt-highlights-and-approvals-20">State of the Field: CGT Highlights and Approvals of 2023</a> </li> <li> <a href="/quick-takes/genetic-testing-and-clinical-trial-eligibility">Genetic Testing and Clinical Trial Eligibility</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/events">Events</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/events/empowering-patients-2025-a-cell-and-gene-therapies">Empowering Patients 2025: A Cell and Gene Therapies Summit</a> </li> <li> <a href="/events/annual-meeting-patient-advocate-offerings">Annual Meeting-Patient Advocate Offerings</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/about-asgct">About</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/about-asgct/overview">Overview</a> </li> <li> <a href="/about-asgct/about-asgct">About ASGCT</a> </li> <li> <a href="/about-asgct/news">ASGCT News</a> </li> <li> <a href="/about-asgct/privacy-policy">Privacy Policy</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/support">Support</a> </li> </ul> <a href="asgct.org" class="px-1 is-external is-hidden-desktop">ASGCT.org</a> </div> </nav> </header> <main class="c-main" id="content"> <section class="c-breadcrumbs"> <div class="container"> <span><a href="/">Home</a> > </span> <a href="/gene-therapy-101" title="Gene & Cell Therapy 101" class="CMSBreadCrumbsLink">Gene & Cell Therapy 101</a> > <span class="CMSBreadCrumbsCurrentItem">Vectors 101</span> </div> </section> <section class="section"> <div class="container"> <div class="columns"> <div class="column is-8"> <h1 class="is-like-h2">Vectors 101</h1> </div> </div> <div class="columns"> <div class="column is-8"> <div class="c-leader"> <div class="c-leader-content"><p>Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell. </p></div> </div> <div class="content mb-6"> <p><iframe allow="accelerometer; autoplay; encrypted-media; gyroscope; picture-in-picture" allowfullscreen="" frameborder="0" height="315" src="https://www.youtube.com/embed/Q6qk6Wh6cXU" width="560"></iframe></p> <p>Gene therapy is a way to treat or prevent a specific disease using genetic material. One common way that researchers have found to accomplish this is by using a vector. Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell. There are four main types of viral vectors (adeno-associated viral, adenoviral, lentiviral, retroviral) each with their own unique characteristics, uses, and limitations.</p> <h2>Using Viruses</h2> <p>Vectors are typically derived from viruses, because viruses have proven to be very efficient at finding their way into cells. In order to make vectors safe to use, all of the viral genes are removed, and the vector is modified to deliver only therapeutic genes. The choice of which viruses to use as vectors is based on three criteria:</p> <ul> <li>How well researchers <strong>understand</strong> the virus</li> <li>How well the virus can <strong>target certain cell types </strong></li> <li>How <strong>safe </strong>the virus is to use</li> </ul> <p><a href="https://asgct.org/global/documents/patient-ed-infographics/sept-launch-website-material/viral_vector_overview-(7).aspx" target="_blank"><img alt="" src="https://asgct.org/getmedia/c191081e-c02d-4712-80eb-5889af3a54d3/Viral-Vector-Overview-infographic.aspx?width=350&height=350" style="width: 350px; height: 350px; float: right; margin: 5px 10px;" /></a>Vectors make use of the shell of the virus, also known as the capsid, to help transport working genes to the target cell. Think of vectors like envelopes being used to deliver a specific message. The envelope allows the message to be delivered, while the message inside provides new instructions to the cell on how to function properly. It might sound backward to use a virus to treat a disease, but the use of vectors is backed by years of research and has been shown to be safe, which is always a top priority.</p> <p>Gene therapy using viral vectors does more than just treat symptoms. Vectors, with the genetic information they carry, can directly target the cause of a disease and change the way a cell functions. In addition, they typically only need to be administered once and are commonly used for rare inherited diseases that have few to no other treatment options available.</p> <p><img alt="" src="https://asgct.org/getmedia/e565f978-555e-4195-a46d-3375d132b53a/Updated-Vectors-Photo.aspx?width=799&height=393" style="width: 799px; height: 393px;" /></p> <h3><strong>Adeno-Associated Viral (AAV) Vectors</strong></h3> <p>Adeno-associated viral vectors, also known as AAVs, are typically used to deliver smaller DNA packages or genes. The size capacity of this vector may be a factor to determine which rare diseases it can target. They’re known to be safe and efficient when used for<em> in vivo</em> gene therapy approaches. <em>In vivo</em> therapy involves injecting a gene therapy into specific parts of the body so that new genetic instructions can be delivered directly to the cells within the body.</p> <p>AAVs are usually <strong>non-integrating</strong>. That means the DNA they carry doesn’t typically insert itself into the cell’s genome. So, if the vector is taken up by a cell that divides, the therapeutic gene won’t be copied with each cell division and may be lost over time, thereby diluting the treatment effect. Because of this, AAVs are commonly used to target non-dividing cells, such as cells in the liver, nervous system, eyes, and skeletal muscles. AAVs can persist in patients for a prolonged period of time—possibly even a lifetime.</p> <p><strong>Innate immunity</strong> is one limitation of AAVs since many people may have had prior exposure to AAVs through natural infections. This means that pre-existing antibodies can disqualify anywhere from 30 to 70 percent of patients from clinical trial participation depending on the type of AAV being used. For these patients, their immune system may attack or destroy the vector before it can deliver its therapeutic package, rendering it useless. In addition, patients will often be limited to a single administration of therapy because they may develop antibodies following the first administration. Testing for innate immunity is becoming more widespread and is used as an exclusion criteria for <a href="/clinical-trials/considering-a-clinical-trial">clinical trial eligibility</a>. For now, scientists are working on a variety of strategies to help combat this challenge such as creating synthetic capsids that would not be attacked by the immune system or by utilizing an enzyme that can split through the antibodies in the short term. These solutions come from knowing there is a short window of time (hours) where the vector is injected and then travels to the target cell and once the vector makes it inside the cell it is protected from antibodies.</p> <h3><strong>Adenoviral Vectors</strong></h3> <p>In some ways, adenoviral vectors are similar to AAV vectors. For instance, they’re most often used to deliver DNA packages into non-dividing cells. However, adenoviral vectors are larger and capable of delivering genetic packages almost eight times the size of AAVs. Adenoviral vectors can cause <strong>strong immune responses</strong>, which result in potentially harmful inflammation throughout the body and decreased effectiveness of the therapy. In recent years, scientists have worked to develop adenoviral vectors that result in milder immune responses in order to deliver larger packages with less risk.</p> <h3><strong>Lentiviral and Retroviral Vectors</strong></h3> <p>Lentiviral and retroviral vectors can carry larger genetic packages of RNA, which is converted into DNA. During this process, the vectors <strong>integrate</strong> into the genome of the target cell, unlike AAVs and adenoviral vectors.</p> <p>The ability to integrate into the cell genome makes lentiviral and retroviral vectors best suited for dividing cells, which are targets of an <em>ex vivo</em> treatment approach. For example, these vectors are used to target T cells, which are a type of immune cell, and stem cells, which are special cells that can develop into many cell types. In contrast to <em>in vivo</em> treatments, <em>ex vivo</em> treatment of cells occurs outside the body. Cells are removed from the body and are cultured on plates allowing them to replicate and expand. Then these cells are treated with vectors and modified with new genetic instructions, such as a working gene. The modified cells are then returned to the body. After that, the treated cells begin to divide and generate new cells. Thanks to integration, the new genetic material is copied into all the new cells and continues to function beyond the original cell.</p> <h2><strong>Other Limitations</strong></h2> <p>Gene therapy using viral vectors carries a lot of potential, but is not without some risk. These examples are are in addition to limitations that have been discussed above including innate immunity and strong immune responses.</p> <p><strong>Off-target effects</strong> are when tissues other than the main target tissue may be affected after administration. Vectors that insert their genetic package into the host genome can potentially integrate at an incorrect location of the genome and cause unintended consequences. In order to maintain as much control over the process as possible, researchers have developed targeting techniques. In addition, they have the ability to monitor the vector’s long-term effects in patients at the insertion site.</p> <p><strong>Manufacturing</strong> is another challenge that is being addressed by professionals in the field. Creating a very large quantity of safe viral vectors requires time, effort, and resources. The complexities of the process add to manufacturing costs and makes it hard to effectively streamline production. Researchers will continue working towards more efficient production methods as more gene therapies are researched and approved for use.</p> <h2><strong>Non-Viral Vectors</strong></h2> <p>Researchers are also developing more non-viral vectors to address (or eliminate) these limitations. Non-viral vectors are cheaper to manufacture than their viral counterparts. They can potentially deliver larger genetic packages, allow for repeated dosing, and make quality control easier. Non-viral vectors also have the benefit of a lowered chance of triggering adverse immune responses.</p> <p>While non-viral vectors hold a lot of promise, scientists simply have less experience with them. Many of the non-viral mechanisms are developing rapidly but challenges still remain, such as carrying capacity and ability to target specific organs or tissues. For now, they can’t be used for<em> in vivo</em> approaches that are common with AAVs and adenoviral vectors.</p> <h2><strong>Gene Therapy and Oncology</strong></h2> <p>The approach to gene therapy is very different when it comes to oncology and cancer treatment. Current vectors cannot treat 100% of cells, so gene addition or specific gene targeting strategies, which are methods used for rare diseases such as adding a working gene, simply aren’t as effective for cancer treatment. However, there are strategies that use gene therapy to treat cancer by enhancing the immune response to cancer cells. These methods vary greatly from rare disease treatment, in which a working gene sits next to a faulty gene. One way to treat cancer is to provide dividing immune cells with new genetic instructions that can turn them into cells that <a href="/disease-treatments/car-t-basics">recognize and kill cancer cells</a>. Another approach is to use vectors that can only multiply rapidly in tumor cells. From there, the vectors with specific instructions to kill those cancer cells are able to do so.</p> </div> </div> <div class="column is-4 pl-4"> <div class="c-nav-local-wrap"> <div class="asideBox"> <h3> Related Pages</h3> <hr class="is-secondary" /> <ul class="c-nav-local-list"> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/gene-therapy-basics">Gene Therapy Basics</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/cell-therapy-basics">Cell Therapy Basics</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/gene-therapy-approaches">Gene Therapy Approaches</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/gene-editing">Gene Editing</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/clinical-trials">Clinical Trials</a> </li> <li class="c-nav-local-item is-active"><a class="c-nav-local-link" href="/gene-therapy-101/vectors-101">Vectors 101</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/covid19-vaccines">COVID-19</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/lunch-learn-gene-therapy-for-sma">Lunch & Learn: Gene Therapy for SMA</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/helpful-websites">Helpful Websites</a> </li> </ul> </div> </div> </div> </div> </div> <div class="lastUpdatedTag"> <p>Last Updated: 01/30/2024</p> </div> </section> </main> <footer class="c-footer"> <div class="c-footer-primary"> <section class="section"> <div class="container"> <div class="columns"> <div class="column c-footer-section"> <p><img alt="" src="/PatientEducation/media/PatientEducation/logo_white.png" /></p> <p>20800 Swenson Dr., Suite 300<br /> Waukesha, WI 53186</p> </div> <div class="column is-two-fifths c-footer-section"> <ul class="c-utility-list"> <li><a href="#">Gene Therapy 101</a></li> <li><a href="#">Disease Treatments</a></li> <li><a href="#">Clinical Trials</a></li> <li><a href="/patient-journey">Patient Journey</a></li> <li><a href="/about-asgct">About</a></li> <li><a href="/support">Support</a></li> <li><a href="https://asgct.org/about/privacy-policy" target="_blank">Privacy Policy</a></li> </ul> </div> <div class="column c-footer-section"> <h3 class="mb-3">Connect with ASGCT</h3> <p class="mb-0"><span>Phone: 414</span>.278.1341</p> <p><a href="mailto:info@asgct.org">info@asgct.org</a></p> <p> <div class="c-social"> <ul class="c-social-list"> <li class="c-social-item"><a href="https://twitter.com/ASGCTherapy" target="_blank" class="c-social-link twitter"><span class="is-sr-only">Twitter</span></a></li> <li class="c-social-item"><a href="https://www.facebook.com/ASGCTherapy" target="_blank" class="c-social-link facebook"><span class="is-sr-only">Facebook</span></a></li> <li class="c-social-item"><a href="https://www.instagram.com/asgctherapy" target="_blank" class="c-social-link instagram"><span class="is-sr-only">Instagram</span></a></li> <li class="c-social-item"><a href="https://www.linkedin.com/company/asgct" target="_blank" class="c-social-link linkedin"><span class="is-sr-only">LinkedIn</span></a></li> <li class="c-social-item"><a href="https://www.youtube.com/user/ASGCT" target="_blank" class="c-social-link youtube"><span class="is-sr-only">YouTube</span></a></li> </ul> </div> </p> </div> </div> </div> </section> </div> <div class="c-footer-secondary"> <section class="section py-4"> <div class="container"> <div class="columns"> <div class="column"> <p class="is-small">© 2024 All rights reserved. American Society of Gene & Cell Therapy.</p> <p class="is-tiny">Web design by <a href="https://www.reasononeinc.com/kentico" target="_blank">Reason One</a>. Powered by <a href="http://www.kentico.com" target="_blank">Kentico</a>.</p> </div> </div> </div> </section> </div> </footer> <div class="CookieConsent"> <span id="ctl00_cookieLawQ_lblText" class="ConsentText">This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.</span> <span class="ConsentButtons"> <input type="submit" name="ctl00$cookieLawQ$btnAllowSpecific" value="OPT OUT" id="ctl00_cookieLawQ_btnAllowSpecific" class="ConsentButton btn btn-default" /> <input type="submit" name="ctl00$cookieLawQ$btnAllowAll" value="ACCEPT" id="ctl00_cookieLawQ_btnAllowAll" class="ConsentButton btn btn-default" /> </span> </div> <script src="/BlueKey/pe_assets/build/main.js" type="text/javascript"></script> <script type="text/javascript"> //<![CDATA[ var callBackFrameUrl='/WebResource.axd?d=nNME_ObE2oIDm535eYM9xWmIMy-HXBVdo54Ly6sA5tAYPS1aA-gFDzodlP9ijTBRgmjStONrH4-ISuRTp7slHiYkACw1&t=638562384440000000'; WebForm_InitCallback();//]]> </script> </form> </body> </html>