{"id":899,"date":"2023-06-13T13:26:17","date_gmt":"2023-06-13T13:26:17","guid":{"rendered":"\/?page_id=899"},"modified":"2023-12-01T13:45:27","modified_gmt":"2023-12-01T13:45:27","slug":"research","status":"publish","type":"page","link":"https:\/\/lab.research.sickkids.ca\/ivakine\/research\/","title":{"rendered":"Research"},"content":{"rendered":"<p><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-1 fusion-flex-container nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-padding-top:25px;--awb-padding-right:25px;--awb-padding-bottom:25px;--awb-padding-left:25px;--awb-background-color:#09254c;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-justify-content-center fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-0 fusion_builder_column_2_3 2_3 fusion-flex-column\" style=\"--awb-bg-blend:overlay;--awb-bg-size:cover;--awb-width-large:66.666666666667%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:0%;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-1 fusion-sep-none fusion-title-center fusion-title-text fusion-title-size-one\" style=\"--awb-text-color:var(--awb-color2);--awb-font-size:64px;\"><h1 class=\"fusion-title-heading title-heading-center fusion-responsive-typography-calculated\" style=\"margin:0;font-size:1em;--fontSize:64;line-height:1.1;\">Research<\/h1><\/div><div class=\"fusion-separator\" style=\"align-self: center;margin-left: auto;margin-right: auto;margin-bottom:30px;width:100%;max-width:100px;\"><div class=\"fusion-separator-border sep-single sep-solid\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-color4);border-color:var(--awb-color4);border-top-width:2px;\"><\/div><\/div><div class=\"fusion-text fusion-text-1\" style=\"--awb-text-color:var(--awb-color1);\"><p style=\"text-align: center;\">A deeper look into our methodologies and techniques<\/p>\n<\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-2 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-padding-top:0px;--awb-padding-right:0px;--awb-padding-left:0px;--awb-margin-top:50px;--awb-margin-bottom:50px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-1 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-bg-color:var(--awb-color1);--awb-bg-color-hover:var(--awb-color1);--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-2\"><blockquote>\n<p>The overarching focus of the Ivakine Lab can be boiled down to two main ideas: 1) understanding the functionality of genetic variants, at scale, through saturation genome editing; and 2) developing novel CRISPR-based and small molecule-based therapeutics to generate precision therapies for rare genetic disorders \u2014 with a focus on neurodegenerative and neurodevelopmental conditions. We are using cutting edge genome editing tools and delivery vehicles to generate and correct disease-causing mutations in cell and animal models.<\/p>\n<\/blockquote>\n<\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-3 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-padding-top:50px;--awb-padding-right:50px;--awb-padding-bottom:50px;--awb-padding-left:50px;--awb-margin-top:0px;--awb-background-color:var(--awb-color2);--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-center fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-2 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:50px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-2 fusion-sep-none fusion-title-text fusion-title-size-two\"><h2 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"margin:0;--fontSize:40;line-height:1.3;\">Development of novel therapeutic approaches for the<br \/>\nnext generation of precision medicines<\/h2><\/div><div class=\"fusion-separator\" style=\"align-self: flex-start;margin-right:auto;width:100%;max-width:200px;\"><div class=\"fusion-separator-border sep-single sep-solid\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-custom_color_1);border-color:var(--awb-custom_color_1);border-top-width:3px;\"><\/div><\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-3 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-padding-top:1.9669999999999987px;--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:1;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:1;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-image-element md-text-align-center\" style=\"--awb-caption-title-font-family:var(--h2_typography-font-family);--awb-caption-title-font-weight:var(--h2_typography-font-weight);--awb-caption-title-font-style:var(--h2_typography-font-style);--awb-caption-title-size:var(--h2_typography-font-size);--awb-caption-title-transform:var(--h2_typography-text-transform);--awb-caption-title-line-height:var(--h2_typography-line-height);--awb-caption-title-letter-spacing:var(--h2_typography-letter-spacing);\"><span class=\" fusion-imageframe imageframe-none imageframe-1 hover-type-none fusion-animated\" data-animationType=\"fadeInLeft\" data-animationDuration=\"2.8\" data-animationOffset=\"top-into-view\"><a href=\"\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy.png\" class=\"fusion-lightbox\" data-rel=\"iLightbox[7191aff96d834a0ab5b]\" data-title=\"Untitled (2) copy\" title=\"Untitled (2) copy\"><img decoding=\"async\" width=\"720\" height=\"504\" alt=\"immunohistochemistry images of brain (cerebellum and hippocampus) and muscle tissue from mice. staining shows DAPI and calbindin positive cells in the cerebellum, NeuN positive cells in the hippocampus, and dystrophin and DAPI in muscle tissue.\" src=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy.png\" data-orig-src=\"\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy.png\" class=\"lazyload img-responsive wp-image-1355\" srcset=\"data:image\/svg+xml,%3Csvg%20xmlns%3D%27http%3A%2F%2Fwww.w3.org%2F2000%2Fsvg%27%20width%3D%27720%27%20height%3D%27504%27%20viewBox%3D%270%200%20720%20504%27%3E%3Crect%20width%3D%27720%27%20height%3D%27504%27%20fill-opacity%3D%220%22%2F%3E%3C%2Fsvg%3E\" data-srcset=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy-200x140.png 200w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy-400x280.png 400w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy-600x420.png 600w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/Untitled-2-copy.png 720w\" data-sizes=\"auto\" data-orig-sizes=\"(max-width: 1024px) 100vw, (max-width: 640px) 100vw, 720px\" \/><\/a><\/span><\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-4 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-padding-right:36px;--awb-padding-left:32.98609999999999px;--awb-padding-right-small:0px;--awb-padding-left-small:0px;--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:3.84%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:3.84%;--awb-width-medium:100%;--awb-order-medium:2;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:2;--awb-spacing-right-small:0%;--awb-spacing-left-small:0%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-3\" style=\"--awb-content-alignment:left;--awb-text-color:var(--awb-custom_color_4);\"><p>Genetic diseases manifest in various ways, some affecting the brain, muscles and several other organs. Disease-causing mutations function similarly. Our lab focuses on a variety of these mutations, including:<\/p>\n<ul>\n<li>simple single nucleotide changes, such as Joubert-like syndrome mutation<\/li>\n<li>small deletions and insertions, such as Tay-Sachs Disease mutation in HEXA<\/li>\n<li>duplications of large DNA fragments, such as MECP2 duplication syndrome<\/li>\n<li>deletions of large genetic regions, such as the exon 52 deletion in the gene that causes Duchenne Muscular Dystrophy<\/li>\n<\/ul>\n<\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-5 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:50px;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:4;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:4;--awb-spacing-right-small:0%;--awb-spacing-left-small:0%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-4\" style=\"--awb-content-alignment:left;--awb-text-color:var(--awb-custom_color_4);\"><p class=\"p1\">A plethora of disease causing mutations, coupled with different organs&#8217; involvement, makes it challenging to develop therapies. We are using a whole arsenal of recently developed genome engineering tools (base editing, prime editing) to first model a mutation in a relevant cell line, analyze it for functional consequences and interrogate various correction strategies. We then develop a relevant model of a disease, and after thorough characterization, we apply our most promising strategies to elicit a therapeutic effect in vivo. We are closely working with regulatory agencies to bring these novel therapies into the clinic.<\/p>\n<\/div><div style=\"text-align:center;\"><a class=\"fusion-button button-flat button-xlarge button-custom fusion-button-default button-1 fusion-button-span-yes fusion-button-default-type\" style=\"--button_accent_color:var(--awb-color1);--button_accent_hover_color:var(--awb-color1);--button_border_hover_color:var(--awb-color1);--button_gradient_top_color:var(--awb-custom_color_2);--button_gradient_bottom_color:var(--awb-custom_color_2);--button_gradient_top_color_hover:var(--awb-custom_color_1);--button_gradient_bottom_color_hover:var(--awb-custom_color_1);--button_margin-top:25px;\" target=\"_self\" href=\"\/ivakine\/projects\/\"><span class=\"fusion-button-text awb-button__text awb-button__text--default\">Learn more about our novel therapeutic approaches<\/span><i class=\"fa-angle-right fas awb-button__icon awb-button__icon--default button-icon-right\" aria-hidden=\"true\"><\/i><\/a><\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-6 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:3.84%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:3.84%;--awb-width-medium:100%;--awb-order-medium:3;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:3;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-image-element \" style=\"text-align:center;--awb-caption-title-font-family:var(--h2_typography-font-family);--awb-caption-title-font-weight:var(--h2_typography-font-weight);--awb-caption-title-font-style:var(--h2_typography-font-style);--awb-caption-title-size:var(--h2_typography-font-size);--awb-caption-title-transform:var(--h2_typography-text-transform);--awb-caption-title-line-height:var(--h2_typography-line-height);--awb-caption-title-letter-spacing:var(--h2_typography-letter-spacing);\"><span class=\" fusion-imageframe imageframe-none imageframe-2 hover-type-none fusion-animated\" data-animationType=\"fadeInRight\" data-animationDuration=\"2.8\" data-animationOffset=\"top-into-view\"><a href=\"\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720.png\" class=\"fusion-lightbox\" data-rel=\"iLightbox[9edb9658434b1534f5e]\"><img decoding=\"async\" width=\"720\" height=\"487\" alt=\"Infographic depicting saturation editing workflow. Workflow images show: epegRNA cloning into lentiviral vectors, virus projection and cell transduction, sample collection and functional assay, and amplicon sequencing\/SNV functional interpretation.\" src=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720.png\" data-orig-src=\"\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720.png\" class=\"lazyload img-responsive wp-image-1431\" srcset=\"data:image\/svg+xml,%3Csvg%20xmlns%3D%27http%3A%2F%2Fwww.w3.org%2F2000%2Fsvg%27%20width%3D%27720%27%20height%3D%27487%27%20viewBox%3D%270%200%20720%20487%27%3E%3Crect%20width%3D%27720%27%20height%3D%27487%27%20fill-opacity%3D%220%22%2F%3E%3C%2Fsvg%3E\" data-srcset=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720-200x135.png 200w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720-400x271.png 400w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720-600x406.png 600w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/figure_1_for_dk-3_720.png 720w\" data-sizes=\"auto\" data-orig-sizes=\"(max-width: 1024px) 100vw, (max-width: 640px) 100vw, 720px\" \/><\/a><\/span><\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-4 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-padding-top:50px;--awb-padding-right:50px;--awb-padding-bottom:50px;--awb-padding-left:50px;--awb-padding-top-small:50px;--awb-padding-right-small:50px;--awb-padding-bottom-small:50px;--awb-padding-left-small:50px;--awb-margin-top:0px;--awb-margin-bottom:100px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-center fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-7 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:50px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-3 fusion-sep-none fusion-title-text fusion-title-size-two\"><h2 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"margin:0;--fontSize:40;line-height:1.3;\">CRISPR and drug-based screens to rescue disease pathogenesis<\/h2><\/div><div class=\"fusion-separator\" style=\"align-self: flex-start;margin-right:auto;width:100%;max-width:200px;\"><div class=\"fusion-separator-border sep-single sep-solid\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-custom_color_1);border-color:var(--awb-custom_color_1);border-top-width:3px;\"><\/div><\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-8 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-image-element \" style=\"text-align:center;--awb-caption-title-font-family:var(--h2_typography-font-family);--awb-caption-title-font-weight:var(--h2_typography-font-weight);--awb-caption-title-font-style:var(--h2_typography-font-style);--awb-caption-title-size:var(--h2_typography-font-size);--awb-caption-title-transform:var(--h2_typography-text-transform);--awb-caption-title-line-height:var(--h2_typography-line-height);--awb-caption-title-letter-spacing:var(--h2_typography-letter-spacing);\"><span class=\" fusion-imageframe imageframe-none imageframe-3 hover-type-none fusion-animated\" data-animationType=\"fadeInLeft\" data-animationDuration=\"2.8\" data-animationOffset=\"top-into-view\"><a href=\"\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail.jpg\" class=\"fusion-lightbox\" data-rel=\"iLightbox[afbc41d479b5a79ef4d]\" data-title=\"crispr-thumbnail\" title=\"crispr-thumbnail\"><img decoding=\"async\" width=\"480\" height=\"480\" alt=\"computer-generated image of CRISPR molecule binding to a strand of DNA\" src=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail.jpg\" data-orig-src=\"\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail.jpg\" class=\"lazyload img-responsive wp-image-65\" srcset=\"data:image\/svg+xml,%3Csvg%20xmlns%3D%27http%3A%2F%2Fwww.w3.org%2F2000%2Fsvg%27%20width%3D%27480%27%20height%3D%27480%27%20viewBox%3D%270%200%20480%20480%27%3E%3Crect%20width%3D%27480%27%20height%3D%27480%27%20fill-opacity%3D%220%22%2F%3E%3C%2Fsvg%3E\" data-srcset=\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail-200x200.jpg 200w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail-400x400.jpg 400w, https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/06\/crispr-thumbnail.jpg 480w\" data-sizes=\"auto\" data-orig-sizes=\"(max-width: 1024px) 100vw, (max-width: 640px) 100vw, 480px\" \/><\/a><\/span><\/div><div class=\"fusion-text fusion-text-5\" style=\"--awb-margin-top:25px;\"><p style=\"text-align: center;\">Image credit: <a href=\"https:\/\/www.biointeractive.org\/classroom-resources\/crispr-cas9-mechanism-applications\" target=\"_blank\" rel=\"noopener\">HHMI BioInteractive<\/a><\/p>\n<\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-9 fusion_builder_column_1_2 1_2 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:50%;--awb-margin-top-large:0px;--awb-spacing-right-large:0%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-6\" style=\"--awb-content-alignment:left;--awb-text-color:var(--awb-custom_color_4);\"><p>We have recently initiated CRISPR-KO and CRISPRa screens to not only gain a deeper insight into disease pathogenesis but to also identify molecular pathways in which modulation may influence disease progression and severity. Complementary, high throughput functional assays are also performed to identify novel therapeutic candidates.<\/p>\n<\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-5 fusion-flex-container nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-padding-top:100px;--awb-padding-right:100px;--awb-padding-bottom:100px;--awb-padding-left:100px;--awb-margin-top:0px;--awb-background-color:#0f2e51;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-justify-content-center fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-10 fusion_builder_column_1_3 1_3 fusion-flex-column fusion-flex-align-self-center\" style=\"--awb-bg-blend:overlay;--awb-bg-size:cover;--awb-width-large:33.333333333333%;--awb-margin-top-large:0px;--awb-spacing-right-large:5.76%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:5.76%;--awb-width-medium:100%;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-flex-justify-content-flex-start fusion-content-layout-column\"><i class=\"fb-icon-element-1 fb-icon-element fontawesome-icon fa-dna fas circle-yes\" style=\"--awb-iconcolor:var(--awb-custom_color_1);--awb-iconcolor-hover:var(--awb-custom_color_1);--awb-circlecolor:#ffffff;--awb-circlecolor-hover:#ffffff;--awb-circlebordercolor-hover:var(--awb-color8);--awb-circlebordersize:1px;--awb-font-size:65.12px;--awb-width:130.24px;--awb-height:130.24px;--awb-line-height:128.24px;--awb-align-self:center;--awb-margin-top:0px;--awb-margin-right:0px;--awb-margin-bottom:50px;--awb-margin-left:0px;\"><\/i><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-11 fusion_builder_column_1_3 1_3 fusion-flex-column fusion-flex-align-self-center\" style=\"--awb-bg-size:cover;--awb-width-large:33.333333333333%;--awb-margin-top-large:0px;--awb-spacing-right-large:18.432%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-7\" style=\"--awb-content-alignment:center;--awb-text-color:var(--awb-color1);\"><h2 style=\"text-align: center\"><span style=\"color: var(--awb-text-color);font-family: var(--awb-text-font-family);font-size: var(--awb-font-size);font-style: var(--awb-text-font-style);font-weight: var(--awb-text-font-weight);letter-spacing: var(--awb-letter-spacing);text-align: var(--awb-content-alignment);text-transform: var(--awb-text-transform);background-color: var(--awb-bg-color-hover)\">Find us<\/span><\/h2>\n<\/div><div class=\"fusion-text fusion-text-8\" style=\"--awb-text-color:var(--awb-color1);\"><p style=\"text-align: center\">Peter Gilgan Centre for Research and Learning<br \/>\n686 Bay Street, Toronto, ON M5G 0A4<\/p>\n<\/div><\/div><\/div><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-12 fusion_builder_column_1_3 1_3 fusion-flex-column fusion-flex-align-self-center\" style=\"--awb-bg-size:cover;--awb-width-large:33.333333333333%;--awb-margin-top-large:0px;--awb-spacing-right-large:5.76%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:5.76%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div style=\"text-align:center;\"><a class=\"fusion-button button-flat button-xlarge button-custom fusion-button-default button-2 fusion-button-default-span fusion-button-default-type\" style=\"--button_accent_color:var(--awb-custom_color_1);--button_border_color:var(--awb-custom_color_1);--button_accent_hover_color:var(--awb-color1);--button_border_hover_color:var(--awb-color1);--button_gradient_top_color:var(--awb-color1);--button_gradient_bottom_color:var(--awb-color1);--button_gradient_top_color_hover:var(--awb-custom_color_1);--button_gradient_bottom_color_hover:var(--awb-custom_color_1);\" target=\"_self\" href=\"\/contact\/\"><span class=\"fusion-button-text awb-button__text awb-button__text--default\">Contact us<\/span><\/a><\/div><\/div><\/div><\/div><\/div>\n<\/p>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":438,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"100-width.php","meta":{"footnotes":""},"class_list":["post-899","page","type-page","status-publish","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.0 (Yoast SEO v27.0) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Research - Ivakine Lab<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/lab.research.sickkids.ca\/ivakine\/research\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Research\" \/>\n<meta property=\"og:url\" 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