BNFo 491/591 
Subcommittee on Genetic Modification
Orientation (Staffers) - Tour: Genetic correction of Usher syndrome
Spring 2019 

Background
Representative Bob Frappuccino (OH) passed on to our committee a letter from a constituent:

I'm a constituent in your district in Upper Sandusky. My sister was born weak in the ears but she manages. Now her eyes are beginning to give out. The doctors say she got all this from our parents (though they were both fine). They call it Usher syndrome. I have to take care of her, and I'm getting in not so good shape myself. It's harder and harder. I don't mean to bother you about our problems, but I was reading about what you in Congress were doing after the Princess Juliana thing, and when my sister's eye doctor gave me an article about Usher's syndrome, I thought maybe it's something Congress can do something about. If you can spend millions of dollars for a road in Alaska that doesn't go anywhere, maybe you can spend something to cure Usher syndrome what people really care about. Oh, here's the article:
Spanish research suggests gene editing may provide a potential treatment for Usher's syndrome.
Euretina Brief (17 Nov 2017)
Thanks for your time. I voted for you.
[Name blocked out]
Rep. Frappuccino accompanied this correspondence with the instructions that we make every effort to look into the matter as soon as possible. I now pass both on to you.

Suggestions for staffers

  • Read the article given by the constituent
     
  • Identify and obtain the research article on which it is based
     
  • It might also help to obtain a review article that can give you an overview about how genome editing might address hearing loss
     
  • Read what you found, understand what you can, and (most important) collect and prioritize concepts you don't understand, those you must grasp to achieve your goal
     
  • Your goal: Prepare for your representative a position paper that describes the prospects for curing Usher syndrome by means of genome editing, including a comprehensible explanation of the molecular mechanism by which it might be done.
     
  • You're unlikely to be able to dash this off. Better, bring your thoughts and especially your prioritized list of problem areas to a discussion with your staffer colleagues scheduled for Tuesday, Feb 5.
     
  • Don't embarrass yourself and your representative by getting stuck. If you encounter an obstacle, either find a way to overcome it, or reach out to a colleague for advice. Make sure you have found the relevant articles by Tuesday and have made progress in figuring out what you need to know. Figuring out the articles themselves is less important (and virtually impossible by Tuesday in any case).