Finding Treatment for a Rare Genetic Disease 

HMS scientists are pursuing three gene therapies with the hope that at least one will treat progressive blindness caused by Usher syndrome type 1F.
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Find out more at hms.harvard.edu/news/pursuing...
00:00 What is Usher syndrome Type 1F?
00:53 Inspiration for the project
02:28 Gene therapy and the challenge of finding a treatment
03:37 Three approaches
05:05 Toward a cure