Attachment -II Page 1 
Some questions to be answered before initiation of gene therapy: 
1. Full description of nature of DNA to be used in gene therapy. 
2. What virus, if any? Full description of the viral construct to be used 
for gene therapy. 
3. What animal studies have been done? Full background and details. 
4. What disease is to be treated? State reasons why this disease selected 
5. Age and sex of patient. Other affected family members? 
6. Methodology of gene transfer to patient? 
7. How is patient monitored? How will patient be assessed to ascertain 
whether gene therapy is working? How will possible general side effects of 
gene therapy be monitored? 
8. Is natural history of disease known so “that assessment of success of 
gene therapy is clear and straightforward? 
9. Can longterm follow-up be assured? Autopsy, if patient dies? 
10. What criteria are used in selecting a specific patient? Where will such 
a patient or patients be found? 
11. How will the protocol be presented to parents, if patient is child? 
12. Consent by child - at what age? 
13. Which hospital will be used? Regular hospital bed? Clinical research 
center bed? 
14. Who supports costs of gene therapy? Research grants (public? private?) 
If private, is gene technology company involved? Will patients have to pay? 
How much? 
15. Are patents involved? 
16. Alternatives to gene therapy? Status and prospects of therapy other 
than gene therapy? 
17. Effect on protocol if patient withdrew from study at various stages of 
investigation? Any plans to diminish possibility of such an event? 
18. How many patients are to be treated? 
19. What professional personnel (nonmedical and medical) is involved? 
Specific qualifications and experience regarding disease to be treated? 
20. Provide details of local IRB review and curriculum vitae of local IRB 
members. 
[9] 
A. G. Motulsky 
