The Ivakine Lab<\/h1><\/div>The Ivakine Lab<\/h1><\/div>Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
The Ivakine Lab<\/h1><\/div>Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
A targeted approach<\/h2><\/div><\/div><\/div>Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.<\/p>\n
We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.<\/p>\n<\/div>
<\/div><\/div><\/div>![\"A](\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/08\/lab-group-photo-scaled.jpg\")
<\/span><\/div><\/div><\/div><\/div><\/div>![](\"data:image\/gif;base64,R0lGODlhAQABAAAAACH5BAEKAAEALAAAAAABAAEAAAICTAEAOw==\")
<\/span><\/div><\/div>Team<\/h2><\/div>Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
<\/div>Meet our team<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
The Ivakine Lab<\/h1><\/div>Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
The Ivakine Lab<\/h1><\/div>Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
A targeted approach<\/h2><\/div><\/div><\/div>Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.<\/p>\n
We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.<\/p>\n<\/div>
<\/div><\/div><\/div><\/span><\/div><\/div><\/div><\/div><\/div><\/span><\/div><\/div>Team<\/h2><\/div>Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
<\/div>Meet our team<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
The Ivakine Lab<\/h1><\/div>Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
A targeted approach<\/h2><\/div><\/div><\/div>Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.<\/p>\n
We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.<\/p>\n<\/div>
<\/div><\/div><\/div><\/span><\/div><\/div><\/div><\/div><\/div><\/span><\/div><\/div>Team<\/h2><\/div>Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
<\/div>Meet our team<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Utilizing novel gene therapies to target rare inherited diseases<\/p>\n<\/div><\/div><\/div><\/div><\/div>
A targeted approach<\/h2><\/div><\/div><\/div>Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.<\/p>\n
We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.<\/p>\n<\/div>
<\/div><\/div><\/div><\/span><\/div><\/div><\/div><\/div><\/div><\/span><\/div><\/div>Team<\/h2><\/div>Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
<\/div>Meet our team<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.<\/p>\n
We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.<\/p>\n<\/div>
<\/span><\/div><\/div><\/div><\/div><\/div>![](\"\/wp-content\/uploads\/sites\/144\/2016\/11\/Disocver_icon.png\"){kind=icon}
<\/span><\/div>Team<\/h2><\/div>Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
<\/div>Meet our team<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Our team facilitates our robust research and passion for gene therapy<\/p>\n<\/div>
![](\"\/wp-content\/uploads\/sites\/144\/2016\/11\/research_icon.png\"){kind=icon}
<\/span><\/div>Research<\/h2><\/div>Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
Read about our research<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Learn more about our methodologies and how we aim to take our research from laboratory to clinic<\/p>\n<\/div>
![](\"\/wp-content\/uploads\/sites\/144\/2016\/11\/Testing_icon.png\"){kind=icon}
<\/span><\/div>Projects<\/h2><\/div>Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>Learn more about our projects<\/span><\/a><\/div><\/div><\/div><\/span><\/div><\/div>Publications<\/h2><\/div>
Our lab focuses on rare genetic disorders, such as Tay-Sachs Disease, Niemann-Pick Type C and MECP2 Duplication Syndrome.<\/p>\n
We also work closely with the Cohn Lab<\/a>, studying Duchenne Muscular Dystrophy<\/p>\n<\/div>![](\"\/wp-content\/uploads\/sites\/144\/2016\/11\/Analysis_icon.png\"){kind=icon}
Publications<\/h2><\/div>
![](\"https:\/\/lab.research.sickkids.ca\/ivakine\/wp-content\/uploads\/sites\/144\/2023\/11\/1234.png\")
<\/span><\/div><\/div><\/div><\/div><\/div>