If you Google “gene therapy clinical trial” you will find that gene therapy has had a resurgence in recent

years.

Pick a clinical trials and answer the following questions about the trial (should be about 1 page answer). Make sure you pick a real scientific trial, not one of the many places that are just fee for service.

1. Who is the sponsor (paying for) the trial?

2. Where (could be multiple locations) is the trial being held?

3. What type and how many volunteers are being recruited?

4. What is the purpose of the trial?

5. What are the primary and secondary endpoints (e.g. what are they measuring?) of the trial

6. Is this an early stage trial to establish safety or are they trying to show efficacy?

7. What do you think are the promises and potential pitfalls of this trial?

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