Splice-switching antisense oligonucleotides as therapeutic drugs

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5001604/

@charset "UTF-8";.required{color:#da452b;margin-left:3px;position:relative;top:-4px;}.ie .site{display:block;}.uploader .action{color:#4f2683;text-decoration:none;font-size:1rem;font-weight:700;}.uploader .action{display:inline-block;line-height:1.25;padding:.875rem 1.25rem;text-align:center;text-decoration:none;-webkit-transition-property:background-color,border-color,color;-o-transition-property:background-color,border-color,color;transition-property:background-color,border-color,color;border-radius:0rem;border:.0625rem solid rgba(0,0,0,0);font-size:1rem;font-weight:700;}.uploader .action{background-color:#f3f3f3;border-color:#8b8e90;color:#231f20 !important;}.uploader .action+.editor-rich-text__tinymce{color:#231f20 !important;}.uploader .action:hover{background-color:#e7e7e7;border-color:#8b8e90;color:#231f20 !important;}.uploader .action:hover+.editor-rich-text__tinymce{color:#231f20 !important;}.pretty-radio,.pretty-checkbox{padding-top:.25rem;padding-bottom:.25rem;padding-left:2.5rem;position:relative;}.pretty-radio label:after,.pretty-checkbox label:after{background:#fff;border:3px solid #e7e7e7;content:"";display:block;height:1.25rem;position:absolute;top:.5rem;left:0;z-index:0;-webkit-transition-property:background-color,border-color,-webkit-box-shadow;transition-property:background-color,border-color,-webkit-box-shadow;transition-property:background-color,border-color,box-shadow;transition-property:background-color,border-color,box-shadow,-webkit-box-shadow;-o-transition-property:background-color,border-color,box-shadow;width:1.25rem;}.pretty-radio [type=radio],.pretty-checkbox [type=checkbox]{-webkit-appearance:none;-moz-appearance:none;appearance:none;cursor:pointer;opacity:0;outline:none;position:absolute;top:0;left:0;bottom:0;z-index:2;width:1.25rem;}.pretty-radio [type=radio]:hover+label:after,.pretty-checkbox [type=checkbox]:hover+label:after{border-color:#4f2683;-webkit-box-shadow:none;box-shadow:none;outline:none;}.pretty-radio [type=radio]:focus+label:after,.pretty-checkbox [type=checkbox]:focus+label:after{border-color:#4f2683;}.pretty-radio [type=radio]:checked+label:after,.pretty-checkbox [type=checkbox]:checked+label:after{border-color:#4f2683;}.pretty-checkbox label:before{content:"";display:block;height:.625rem;border:solid #4f2683;border-width:0 .125rem .125rem 0;opacity:0;position:absolute;top:.75rem;left:.5rem;z-index:1;-webkit-transform:rotate(45deg);-ms-transform:rotate(45deg);transform:rotate(45deg);-webkit-transition-duration:100ms;-o-transition-duration:100ms;transition-duration:100ms;-webkit-transition-property:background-color,border-color box-shadow;-o-transition-property:background-color,border-color box-shadow;transition-property:background-color,border-color box-shadow;width:.3125rem;}.pretty-checkbox label:after{border-radius:.3125rem;}.pretty-checkbox [type=checkbox]:checked+label:after{background:#fff;}.pretty-checkbox [type=checkbox]:checked+label:before{opacity:1;}.pretty-radio [type=radio]:checked+label:after{background:#4f2683;-webkit-box-shadow:inset 0 0 0 .1875rem #fff;box-shadow:inset 0 0 0 .1875rem #fff;opacity:1;}.pretty-radio label:after{border-radius:50%;}hr{-webkit-box-sizing:content-box;box-sizing:content-box;height:0;overflow:visible;}pre{font-family:monospace,monospace;font-size:1em;}abbr[title]{border-bottom:none;text-decoration:underline;text-decoration:underline dotted;-webkit-text-decoration:underline dotted;}b,strong{font-weight:bolder;}code,kbd,samp{font-family:monospace,monospace;font-size:1em;}small{font-size:80%;}sub,sup{font-size:75%;line-height:0;position:relative;vertical-align:baseline;}sub{bottom:-.25em;}sup{top:-.5em;}optgroup,select,textarea{font-family:inherit;font-size:100%;line-height:1.15;margin:0;}select{text-transform:none;}[type=button],[type=reset]{-webkit-appearance:button;}[type=button]::-moz-focus-inner,[type=reset]::-moz-focus-inner{border-style:none;padding:0;}[type=button]:-moz-focusring,[type=reset]:-moz-focusring{outline:1px dotted ButtonText;}fieldset{padding:.35em .75em .625em;}legend{-webkit-box-sizing:border-box;box-sizing:border-box;color:inherit;display:table;max-width:100%;padding:0;white-space:normal;}progress{vertical-align:baseline;}textarea{overflow:auto;}[type=checkbox],[type=radio]{-webkit-box-sizing:border-box;box-sizing:border-box;padding:0;}[type=number]::-webkit-inner-spin-button,[type=number]::-webkit-outer-spin-button{height:auto;}[type=search]{-webkit-appearance:textfield;outline-offset:-2px;}[type=search]::-webkit-search-decoration{-webkit-appearance:none;}details{display:block;}summary{display:list-item;}[hidden]{display:none;}body.noscroll{position:fixed;width:100%;}blockquote{border-left:solid .3125rem #4f2683;margin:0;padding-left:4.6875rem;position:relative;}blockquote::before{color:#4f2683;content:"";font-family:"dashicons";font-size:3.75rem;line-height:1;position:absolute;top:-.3125rem;left:.3125rem;}blockquote p{color:#4f2683;font-size:1.5rem;line-height:1.41;margin-bottom:.9375rem;}blockquote cite{color:#8b8e90;font-style:normal;font-weight:700;}figure{margin-top:.5rem;margin-right:0;margin-left:0;}figcaption{color:#4f2683;font-size:.875rem;letter-spacing:1.17px;line-height:1.125rem;}[type=color],[type=date],[type=datetime],[type=datetime-local],[type=email],[type=month],[type=number],[type=password],[type=search],[type=tel],[type=time],[type=url],[type=week],textarea,select,.uploader .filename{-webkit-appearance:none;display:block;width:100%;background-color:#fff;border:.0625rem solid #e7e7e7;border-radius:0rem;color:#231f20;font-size:1rem;height:3.125rem;padding:.625rem 1rem;}[type=color]:focus,[type=date]:focus,[type=datetime]:focus,[type=datetime-local]:focus,[type=email]:focus,[type=month]:focus,[type=number]:focus,[type=password]:focus,[type=search]:focus,[type=tel]:focus,[type=text]:focus,[type=time]:focus,[type=url]:focus,[type=week]:focus,textarea:focus,select:focus,.uploader .filename:focus{border:.0625rem solid #4f2683;outline:none;}input:not([type=checkbox]):not([type=radio])+label,textarea+label,select+label{color:#585b5d;font-size:.875rem !important;margin-top:.3125rem;margin-bottom:.625rem;}textarea::-webkit-input-placeholder{color:#585b5d;}textarea::-moz-placeholder{color:#585b5d;}textarea:-ms-input-placeholder{color:#585b5d;}textarea::-ms-input-placeholder{color:#585b5d;}input::placeholder,textarea::placeholder{color:#585b5d;}textarea{height:auto;}select{padding-right:2.1875rem;-webkit-appearance:none;-moz-appearance:none;appearance:none;-webkit-backface-visibility:hidden;backface-visibility:hidden;}select::-ms-expand{display:none;}select:not([multiple]){background-image:url("data:image/svg+xml,%3Csvg width=\'999\' height=\'583\' viewBox=\'0 0 999 583\' xmlns=\'http://www.w3.org/2000/svg\'%3E%3Cpath d=\'M998.542 83.05c0 8.667-3.333 16.334-10 23l-466 466c-6.666 6.667-14.333 10-23 10-8.666 0-16.333-3.333-23-10l-466-466c-6.666-6.666-10-14.333-10-23 0-8.666 3.334-16.333 10-23l50-50c6.667-6.666 14.334-10 23-10 8.667 0 16.334 3.334 23 10l393 393 393-393c6.667-6.666 14.334-10 23-10 8.667 0 16.334 3.334 23 10l50 50c6.667 6.667 10 14.334 10 23z\' fill-rule=\'nonzero\' fill=\'%234f2683\'/%3E%3C/svg%3E");background-position:right 16px center;background-repeat:no-repeat;background-size:11px auto;}select[multiple]{height:auto;padding:0;}select[multiple] option{padding:.625rem 1rem;}.uploader{cursor:pointer;overflow:hidden;position:relative;height:3.125rem;}.uploader input{cursor:pointer;height:100%;opacity:0;position:absolute;top:0;right:0;bottom:0;left:0;z-index:2;width:100%;}.uploader .filename{cursor:default;overflow:hidden;padding:.625rem 1rem;padding-right:7.75rem;position:absolute;top:0;left:0;bottom:0;-o-text-overflow:ellipsis;text-overflow:ellipsis;-webkit-transition-property:border-color,background,color;-o-transition-property:border-color,background,color;transition-property:border-color,background,color;white-space:nowrap;width:100%;}.uploader .action{border:.0625rem solid #e7e7e7;border-top-right-radius:0rem;border-bottom-right-radius:0rem;border-top-left-radius:0;border-bottom-left-radius:0;font-size:1rem;line-height:1.4;position:absolute;top:0;right:0;bottom:0;-webkit-transition-property:border-color,background,color;-o-transition-property:border-color,background,color;transition-property:border-color,background,color;}[type=submit]{cursor:pointer;}button:active span,button:focus span{position:relative;top:0;left:0;}select:not([multiple]).nitro-lazy{background-image:none !important;}[type=checkbox],[type=radio]{display:inline-block;height:auto;width:auto;}input[type=search]{border-radius:0;-webkit-border-radius:0;}.h1{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#231f20;font-size:3.25em;font-weight:900;line-height:1.1538461538;margin:1.5em 0 1em;letter-spacing:-.87px;}.h1:first-child{margin-top:0;}.h1:last-child{margin-bottom:0;}h2,.h2{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#231f20;font-size:2.25em;font-weight:900;line-height:1.1111111111;margin:1em 0 .5em;}h2:first-child,.h2:first-child{margin-top:0;}h2:last-child,.h2:last-child{margin-bottom:0;}h3,.h3{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#231f20;font-size:1.875em;font-style:#231f20;font-weight:900;line-height:1.4666666667;margin:1em 0 .5em;letter-spacing:-.58px;}h3:first-child,.h3:first-child{margin-top:0;}h3:last-child,.h3:last-child{margin-bottom:0;}h4,.h4{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#231f20;font-size:1.625em;font-weight:900;line-height:1.4615384615;margin:1em 0 .5em;}h4:first-child,.h4:first-child{margin-top:0;}h4:last-child,.h4:last-child{margin-bottom:0;}h5,.h5{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#4f2683;font-size:1.3125em;font-weight:700;line-height:1.4285714286;margin:1em 0 .5em;letter-spacing:-.7px;}h5:first-child,.h5:first-child{margin-top:0;}h5:last-child,.h5:last-child{margin-bottom:0;}h6,.h6{font-family:"Gotham",Helvetica,Arial,sans-serif;word-break:break-word;color:#82c341;font-size:1em;font-weight:900;line-height:1.125;margin:2em 0 1em;letter-spacing:1px;}h6:first-child,.h6:first-child{margin-top:0;}h6:last-child,.h6:last-child{margin-bottom:0;}h2,.h2,h3,.h3,h6,.h6{text-transform:uppercase;}.h2{word-break:break-word;}.h6{margin-bottom:0;}.h3-alt{color:#4f2683;font-family:"Gotham",Helvetica,Arial,sans-serif;font-weight:normal;font-size:1.625rem;line-height:2.25rem;text-transform:none;margin-bottom:0;word-break:break-word;}hr{background:#8b8e90;border:none;height:.0625rem;margin-top:1.875rem;margin-bottom:1.875rem;}.ie .wp-block-image img{height:100%;}strong,b{font-weight:700;}em,var{font-style:italic;}a:hover{text-decoration:none;}a:hover::after{text-decoration:underline;}a:hover::after{text-decoration:none;}.hover-border a{display:inline-block;font-size:.875rem;font-weight:bold;margin-top:3.125rem;text-decoration:none;text-transform:uppercase;width:100%;}.hover-border a::before{border-top:1px solid rgba(79,38,131,.3);content:"";display:block;height:1px;padding-bottom:1.875rem;-webkit-transition-property:width;-o-transition-property:width;transition-property:width;-webkit-transition-duration:.5s;-o-transition-duration:.5s;transition-duration:.5s;width:15.625rem;}.hover-border a:hover::before{width:100%;}.hover-border a::after{content:"";font-family:"FontAwesome";padding-left:.875rem;}a.disabled{color:#231f20 !important;text-decoration:none;}a.disabled:hover{color:#231f20;}ol{list-style:none;margin:1.25em 0;padding-left:0;}ul.has-yellow-list li::before,ol.has-yellow-list li::before{color:#fec21b;}ul.left-indent,ol.left-indent{margin-left:1.875rem;}ul.consecutive,ol.consecutive{counter-reset:auto;}ol:first-child{margin-top:0;}ol:last-child{margin-bottom:0;}.editor-styles ul,.editor-styles ol{overflow:hidden;}ol[start="2"]{counter-reset:li 1;}ol[start="3"]{counter-reset:li 2;}ol[start="4"]{counter-reset:li 3;}ol[start="5"]{counter-reset:li 4;}ol[start="6"]{counter-reset:li 5;}ol[start="7"]{counter-reset:li 6;}ol[start="8"]{counter-reset:li 7;}ol[start="9"]{counter-reset:li 8;}ol[start="10"]{counter-reset:li 9;}ol[start="11"]{counter-reset:li 10;}ol[start="12"]{counter-reset:li 11;}ol[start="13"]{counter-reset:li 12;}ol[start="14"]{counter-reset:li 13;}ol[start="15"]{counter-reset:li 14;}ol[start="16"]{counter-reset:li 15;}ol[start="17"]{counter-reset:li 16;}ol[start="18"]{counter-reset:li 17;}ol[start="19"]{counter-reset:li 18;}ol[start="20"]{counter-reset:li 19;}ol[start="21"]{counter-reset:li 20;}ol[start="22"]{counter-reset:li 21;}ol[start="23"]{counter-reset:li 22;}ol[start="24"]{counter-reset:li 23;}ol[start="25"]{counter-reset:li 24;}li li:first-child{padding-top:.5rem;}li li:before{color:#82c341;display:inline-block;position:absolute;width:.4375rem;}ul ul,ul ol,ol ul,ol ol{padding-left:0;margin-top:0;}ol{counter-reset:li;}ol li+li{margin-top:1.25rem;}ol li:before{content:"." counter(li);counter-increment:li;direction:rtl;font-weight:400;left:0;text-align:right;}dt{font-weight:500;margin-bottom:.125rem;}dd{margin-bottom:.5rem;}.two-column li,.three-column li,.four-column li{-webkit-column-break-inside:avoid;-moz-column-break-inside:avoid;break-inside:avoid;}@media (min-width: 769px){.two-column,.three-column,.four-column{-webkit-column-gap:1.875rem;-moz-column-gap:1.875rem;column-gap:1.875rem;}}@media (min-width: 769px){.two-column{-webkit-column-count:2;-moz-column-count:2;column-count:2;}}@media (min-width: 769px){.three-column{-webkit-column-count:3;-moz-column-count:3;column-count:3;}}.p{font-weight:400;font-weight:400;font-size:1em;line-height:1.875;margin:1em 0;}p:first-child,.p:first-child{margin-top:0;}p:last-child,.p:last-child{margin-bottom:0;}.p-sm{font-weight:400;font-size:.875em;line-height:1.5714285714;margin:1em 0;}.p-sm:first-child{margin-top:0;}.p-sm:last-child{margin-bottom:0;}.p-lg{font-weight:400;font-size:1.125em;line-height:1.6666666667;margin:1em 0;}.p-lg:first-child{margin-top:0;}.p-lg:last-child{margin-bottom:0;}.p-xl{font-size:1.5625rem;line-height:2.25rem;}.wsp-pages-list{padding-left:0;}table{border-collapse:collapse;margin:1.875rem 0;width:100%;}th,td{border-right:none;}th,td[data-head]:before{font-weight:700;text-align:left;}th{padding:1rem 1.25rem;}td{padding:.625rem 1.25rem;}thead tr{background:#4f2683;color:#fff;letter-spacing:.03125rem;}thead th{border-color:rgba(255,255,255,.1);line-height:1.3333333;vertical-align:middle;}td[data-head]:before{color:#4f2683;font-size:.875rem;padding:0 .625rem;}tr:nth-child(even){background:#e7e7e7;}img.wp-smiley,img.emoji{display:inline !important;border:none !important;box-shadow:none !important;height:1em !important;width:1em !important;margin:0 .07em !important;vertical-align:-.1em !important;background:none !important;padding:0 !important;}.safe-svg-cover{text-align:center;}.safe-svg-cover .safe-svg-inside{display:inline-block;max-width:100%;}.safe-svg-cover svg{height:100%;max-height:100%;max-width:100%;width:100%;}.wp-block-button__link{color:#fff;background-color:#32373c;border-radius:9999px;box-shadow:none;text-decoration:none;padding:calc(.667em + 2px) calc(1.333em + 2px);font-size:1.125em;}.wp-block-file__button{background:#32373c;color:#fff;text-decoration:none;}body .is-layout-flow > .alignleft{float:left;margin-inline-start:0;margin-inline-end:2em;}body .is-layout-flow > .alignright{float:right;margin-inline-start:2em;margin-inline-end:0;}body .is-layout-flow > .aligncenter{margin-left:auto !important;margin-right:auto !important;}body .is-layout-constrained > .alignleft{float:left;margin-inline-start:0;margin-inline-end:2em;}body .is-layout-constrained > .alignright{float:right;margin-inline-start:2em;margin-inline-end:0;}body .is-layout-constrained > .aligncenter{margin-left:auto !important;margin-right:auto !important;}body .is-layout-constrained > :where(:not(.alignleft):not(.alignright):not(.alignfull)){max-width:var(--wp--style--global--content-size);margin-left:auto !important;margin-right:auto !important;}body .is-layout-constrained > .alignwide{max-width:var(--wp--style--global--wide-size);}body .is-layout-flex{display:flex;}body .is-layout-flex{flex-wrap:wrap;align-items:center;}body .is-layout-flex > *{margin:0;}body .is-layout-grid{display:grid;}body .is-layout-grid > *{margin:0;}.has-black-color{color:var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-color{color:var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-color{color:var(--wp--preset--color--white) !important;}.has-pale-pink-color{color:var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-color{color:var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-color{color:var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-color{color:var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-color{color:var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-color{color:var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-color{color:var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-color{color:var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-color{color:var(--wp--preset--color--vivid-purple) !important;}.has-black-background-color{background-color:var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-background-color{background-color:var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-background-color{background-color:var(--wp--preset--color--white) !important;}.has-pale-pink-background-color{background-color:var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-background-color{background-color:var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-background-color{background-color:var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-background-color{background-color:var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-background-color{background-color:var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-background-color{background-color:var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-background-color{background-color:var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-background-color{background-color:var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-background-color{background-color:var(--wp--preset--color--vivid-purple) !important;}.has-black-border-color{border-color:var(--wp--preset--color--black) !important;}.has-cyan-bluish-gray-border-color{border-color:var(--wp--preset--color--cyan-bluish-gray) !important;}.has-white-border-color{border-color:var(--wp--preset--color--white) !important;}.has-pale-pink-border-color{border-color:var(--wp--preset--color--pale-pink) !important;}.has-vivid-red-border-color{border-color:var(--wp--preset--color--vivid-red) !important;}.has-luminous-vivid-orange-border-color{border-color:var(--wp--preset--color--luminous-vivid-orange) !important;}.has-luminous-vivid-amber-border-color{border-color:var(--wp--preset--color--luminous-vivid-amber) !important;}.has-light-green-cyan-border-color{border-color:var(--wp--preset--color--light-green-cyan) !important;}.has-vivid-green-cyan-border-color{border-color:var(--wp--preset--color--vivid-green-cyan) !important;}.has-pale-cyan-blue-border-color{border-color:var(--wp--preset--color--pale-cyan-blue) !important;}.has-vivid-cyan-blue-border-color{border-color:var(--wp--preset--color--vivid-cyan-blue) !important;}.has-vivid-purple-border-color{border-color:var(--wp--preset--color--vivid-purple) !important;}.has-vivid-cyan-blue-to-vivid-purple-gradient-background{background:var(--wp--preset--gradient--vivid-cyan-blue-to-vivid-purple) !important;}.has-light-green-cyan-to-vivid-green-cyan-gradient-background{background:var(--wp--preset--gradient--light-green-cyan-to-vivid-green-cyan) !important;}.has-luminous-vivid-amber-to-luminous-vivid-orange-gradient-background{background:var(--wp--preset--gradient--luminous-vivid-amber-to-luminous-vivid-orange) !important;}.has-luminous-vivid-orange-to-vivid-red-gradient-background{background:var(--wp--preset--gradient--luminous-vivid-orange-to-vivid-red) !important;}.has-very-light-gray-to-cyan-bluish-gray-gradient-background{background:var(--wp--preset--gradient--very-light-gray-to-cyan-bluish-gray) !important;}.has-cool-to-warm-spectrum-gradient-background{background:var(--wp--preset--gradient--cool-to-warm-spectrum) !important;}.has-blush-light-purple-gradient-background{background:var(--wp--preset--gradient--blush-light-purple) !important;}.has-blush-bordeaux-gradient-background{background:var(--wp--preset--gradient--blush-bordeaux) !important;}.has-luminous-dusk-gradient-background{background:var(--wp--preset--gradient--luminous-dusk) !important;}.has-pale-ocean-gradient-background{background:var(--wp--preset--gradient--pale-ocean) !important;}.has-electric-grass-gradient-background{background:var(--wp--preset--gradient--electric-grass) !important;}.has-midnight-gradient-background{background:var(--wp--preset--gradient--midnight) !important;}.has-small-font-size{font-size:var(--wp--preset--font-size--small) !important;}.has-medium-font-size{font-size:var(--wp--preset--font-size--medium) !important;}.has-large-font-size{font-size:var(--wp--preset--font-size--large) !important;}.has-x-large-font-size{font-size:var(--wp--preset--font-size--x-large) !important;}.wp-block-navigation a:where(:not(.wp-element-button)){color:inherit;}.wp-block-pullquote{font-size:1.5em;line-height:1.6;}.mt-0{margin-top:0 !important;}.mb-0{margin-bottom:0 !important;}.pt-0{padding-top:0 !important;}.pb-0{padding-bottom:0 !important;}