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.
Version that includes an audio-description: • Finding Treatment for a Rare Genetic ...
Find out more at https://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
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