Goal 3: Advance Translational Research

Gene therapy in HLBS disorders

How can we utilize gene therapy to cure or ameliorate HLBS disorders?

Tags (Keywords associated with the idea)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Long term benefit for disorders

Challenge in application to diverse disorders

Feasibility and challenges of addressing this CQ or CC :

Technology exists for many disorders.

Balance between risk for long term benefit vs. current therapy


-4 net votes
7 up votes
11 down votes
Idea No. 497