{"@context":"http://iiif.io/api/presentation/3/context.json","id":"https://ualberta.aviaryplatform.com/iiif/x921c1wp9k/manifest","type":"Manifest","label":{"en":["S1E2: The Power of N-of-1"]},"logo":"https://d9jk7wjtjpu5g.cloudfront.net/organizations/logo_images/000/000/128/original/UA_Logo_WHT_RGB_%281%29.png?1725471982","metadata":[{"label":{"en":["Agent"]},"value":{"en":["Tetro, Jason (Creator)","Shahid, Nayiar (Host)","Wilton-Clark, Harry (Sam) (Interviewee)","Tetro, Jason (Producer)"]}},{"label":{"en":["Date"]},"value":{"en":["2026-05-12 (Issued)"]}},{"label":{"en":["Language"]},"value":{"en":["English"]}},{"label":{"en":["Description"]},"value":{"en":["\u003cp\u003eWhat happens when the patient sitting across from you is the only person in the world with their specific condition? In traditional medicine, that's a problem. For Harry Wilton-Clark, it's the whole point.\u003c/p\u003e\r\n\u003cp\u003eIn this episode of Science at the Fifty-Third Degree, host Dr. Nayiar Shahid talks with Harry Wilton-Clark, a researcher at the University of Alberta's Women and Children's Health Research Institute, about a quiet revolution happening at the edges of medicine — the rise of N-of-1 therapies. These are treatments designed not for thousands of patients, but for one.\u003c/p\u003e\r\n\u003cp\u003eHarry's work focuses on Duchenne muscular dystrophy, a rare and fatal disease that strikes children. Using a technique called exon skipping therapy, his lab designs what he calls \"genetic band-aids\" — molecules that bind to a specific mutation in a patient's DNA and hide it from the body, allowing it to function more normally. The therapy isn't built for a population. It's built for a child.\u003c/p\u003e\r\n\u003cp\u003eThis episode covers:\u003c/p\u003e\r\n\u003cp\u003eWhat Duchenne muscular dystrophy is and why it's so difficult to treat\u003c/p\u003e\r\n\u003cp\u003e\u003cbr\u003eHow exon skipping therapy works — and what a \"genetic band-aid\" actually does\u003c/p\u003e\r\n\u003cp\u003e\u003cbr\u003eWhy traditional clinical trials fail rare disease patients — and what N-of-1 trials offer instead\u003c/p\u003e\r\n\u003cp\u003e\u003cbr\u003eHow patient families and advocacy organizations are driving research breakthroughs\u003c/p\u003e\r\n\u003cp\u003e\u003cbr\u003eWhat it felt like when a mouse that wouldn't run — finally ran\u003c/p\u003e\r\n\u003cp\u003e\u003cbr\u003eWhy a single patient's treatment could become a master key for thousands of others\u003cbr\u003eHarry recently represented Canada at the Falling Walls Lab in Berlin — one of 100 researchers selected from around the world to present work breaking the boundaries of science's biggest problems. The wall he chose to break: the one standing between a child and a cure.\u003c/p\u003e\r\n\u003cp\u003e \u003c/p\u003e (Summary)","\u003cp\u003eNote: Science at the Fifty-Third Degree is produced by the University of Alberta Basic Science Administrative Team and available on the Aviary platform and wherever you listen to podcasts. Questions or feedback? Reach us at \u003ca title=\"basicsci@ualberta.ca\" href=\"mailto:basicsci@ualberta.ca\"\u003ebasicsci@ualberta.ca\u003c/a\u003e.\u003c/p\u003e (General)"]}},{"label":{"en":["Subject"]},"value":{"en":["Duchenne muscular dystrophy (Topical)","rare disease (Topical)","N-of-1 trial (Topical)","exon skipping therapy (Topical)","antisense oligonucleotides (Topical)","personalized medicine (Topical)","precision medicine (Topical)","genetic therapy (Topical)","pediatric research (Topical)","University of Alberta (Topical)","WCHRI (Topical)","science podcast (Topical)","Canadian science (Topical)","rare disease treatment (Topical)","Falling Walls Lab (Topical)","Edmonton, Alberta Canada (Geographic)"]}},{"label":{"en":["Rights Statement"]},"value":{"en":["\u003cp\u003e\u003ca title=\"Creative Commons Attribution-NonCommercial 4.0 International\" href=\"https://creativecommons.org/licenses/by-nc/4.0/\"\u003eCreative Commons Attribution-NonCommercial 4.0 International\u003c/a\u003e\u003c/p\u003e"]}}],"summary":{"en":["\u003cp\u003eWhat happens when the patient sitting across from you is the only person in the world with their specific condition? In traditional medicine, that's a problem. For Harry Wilton-Clark, it's the whole point.\u003c/p\u003e\r\n\u003cp\u003eIn this episode of Science at the Fifty-Third Degree, host Dr. Nayiar Shahid talks with Harry Wilton-Clark, a researcher at the University of Alberta's Women and Children's Health Research Institute, about a quiet revolution happening at the edges of medicine \u0026mdash; the rise of N-of-1 therapies. These are treatments designed not for thousands of patients, but for one.\u003c/p\u003e\r\n\u003cp\u003eHarry's work focuses on Duchenne muscular dystrophy, a rare and fatal disease that strikes children. Using a technique called exon skipping therapy, his lab designs what he calls \"genetic band-aids\" \u0026mdash; molecules that bind to a specific mutation in a patient's DNA and hide it from the body, allowing it to function more normally. The therapy isn't built for a population. It's built for a child.\u003c/p\u003e\r\n\u003cp\u003eThis episode covers:\u003c/p\u003e\r\n\u003cp\u003eWhat Duchenne muscular dystrophy is and why it's so difficult to treat\u003c/p\u003e\r\n\u003cp\u003e\u003cbr /\u003eHow exon skipping therapy works \u0026mdash; and what a \"genetic band-aid\" actually does\u003c/p\u003e\r\n\u003cp\u003e\u003cbr /\u003eWhy traditional clinical trials fail rare disease patients \u0026mdash; and what N-of-1 trials offer instead\u003c/p\u003e\r\n\u003cp\u003e\u003cbr /\u003eHow patient families and advocacy organizations are driving research breakthroughs\u003c/p\u003e\r\n\u003cp\u003e\u003cbr /\u003eWhat it felt like when a mouse that wouldn't run \u0026mdash; finally ran\u003c/p\u003e\r\n\u003cp\u003e\u003cbr /\u003eWhy a single patient's treatment could become a master key for thousands of others\u003cbr /\u003eHarry recently represented Canada at the Falling Walls Lab in Berlin \u0026mdash; one of 100 researchers selected from around the world to present work breaking the boundaries of science's biggest problems. The wall he chose to break: the one standing between a child and a cure.\u003c/p\u003e\r\n\u003cp\u003e\u0026nbsp;\u003c/p\u003e","\u003cp\u003eNote: Science at the Fifty-Third Degree is produced by the University of Alberta Basic Science Administrative Team and available on the Aviary platform and wherever you listen to podcasts. Questions or feedback? Reach us at \u003ca title=\"basicsci@ualberta.ca\" href=\"mailto:basicsci@ualberta.ca\" target=\"_blank\" rel=\"noopener\"\u003ebasicsci@ualberta.ca\u003c/a\u003e.\u003c/p\u003e"]},"requiredStatement":{"label":{"en":["Attribution"]},"value":{"en":["\u003cp\u003e\u003ca title=\"Creative Commons Attribution-NonCommercial 4.0 International\" href=\"https://creativecommons.org/licenses/by-nc/4.0/\" target=\"_blank\" rel=\"noopener\"\u003eCreative Commons Attribution-NonCommercial 4.0 International\u003c/a\u003e\u003c/p\u003e"]}},"provider":[{"id":"https://ualberta.aviaryplatform.com/aboutus","type":"Agent","label":{"en":["University of Alberta Library"]},"homepage":[{"id":"https://ualberta.aviaryplatform.com/","type":"Text","label":{"en":["University of Alberta Library"]},"format":"text/html"}],"logo":[{"id":"https://d9jk7wjtjpu5g.cloudfront.net/organizations/logo_images/000/000/128/original/UA_Logo_WHT_RGB_%281%29.png?1725471982","type":"Image"}]}],"thumbnail":[{"id":"https://d9jk7wjtjpu5g.cloudfront.net/collections/default_thumbs/000/003/684/small/Science-at-the-Fifty-Third-Degree-16x9.png?1778098300","type":"Image","format":"image/png"}],"items":[{"id":"https://ualberta.aviaryplatform.com/collections/3684/collection_resources/170065/file/308945","type":"Canvas","label":{"en":["Media File 1 of 1 - The-Power-of-N-of-1.mp3"]},"duration":723.216,"width":640,"height":40,"thumbnail":[{"id":"https://d9jk7wjtjpu5g.cloudfront.net/collections/default_thumbs/000/003/684/small/Science-at-the-Fifty-Third-Degree-16x9.png?1778098300","type":"Image","format":"image/png"}],"items":[{"id":"https://ualberta.aviaryplatform.com/collections/3684/collection_resources/170065/file/308945/content/1","type":"AnnotationPage","items":[{"id":"https://ualberta.aviaryplatform.com/collections/3684/collection_resources/170065/file/308945/content/1/annotation/1","type":"Annotation","motivation":"painting","body":{"id":"https://aviary-p-ualberta.s3.wasabisys.com/collection_resource_files/resource_files/000/308/945/original/The-Power-of-N-of-1.mp3?1778191923","type":"Audio","format":"audio/mpeg","duration":723.216,"width":640,"height":40},"target":"https://ualberta.aviaryplatform.com/collections/3684/collection_resources/170065/file/308945","metadata":[]}]}],"annotations":[]}]}