M.J. Welsh and A.E. Smith, RAC Application 
testing for transfer to other tissues including the gonads in hamsters, and the precautions we 
take, we believe the risk to offspring of health care personnel is exceedingly low. 
B.5. Qualifications of investigators, adequacy of laboratory and clinical facilities. 
Indicate the relevant training and experience of the personnel who will be involved in 
the preclinical studies and clinical administration of recombinant DNA. In addition, 
please describe the laboratory and clinical facilities where the proposed study will he 
performed. 
B.5.a. What professional personnel (medical and nonmedical) will he involved in the 
proposed study and what is their relevant expertise? Please provide curricula vitae of 
key professional personnel ( see Section III-E). 
Drs. Michael J. Welsh and Alan E. Smith and their collaborators have published 
extensively on CF and CFTR (1,7,42,62,147,148). Their work on CFTR showed that 
expression of CFTR complemented the CF defect (9,22), elucidated the function of CFTR 
(24,33-35,45-47,149), and explained how mutations in CFTR cause a loss of function 
(32,48,57,58,60). 
Dr. Alan E. Smith is Senior. Vice President - Research at Genzyme Corporation, and is 
widely experienced in molecular biology and animal virology. Dr. Smith has published 
extensively on picomaviruses, arboviruses, retroviruses and papovaviruses and from 1977- 
1981 was on the Editorial Board of Journal of Virology. Genzyme manufactures and sells 
Ceredase, a drug used to treat Gauchers Disease. Genzyme has also developed the 
recombinant proteins, thyroid stimulating hormone and glucocerebrosidase, that are 
presently in clinical trials. Genzyme will produce and test the Ad2/CFTR- 1 vector. 
Procedures involving the patient will be performed by Drs Michael J. Welsh, M.D., Joseph 
Zabner, M.D., and Scott M. Graham, M.D. Each has substantial clinical experience, 
including the care of patients with communicable, respiratory diseases. In addition. Dr. 
Scott M. Graham, currently performs the majority of the sinus surgery at the University of 
Iowa and is highly skilled in the diagnosis and treatment of disorders that affect the nasal 
mucosa. For the last three years, Dr. Graham has been course director for an 
internationally attended course in Advanced Techniques in Endoscopic Sinus Surgery. The 
nurses and caregivers in the Clinical Research Center are highly skilled at accomplishing 
complex experimental protocols and have experience in caring for patients in isolation. 
Copies of the investigator's curricula vitae are located in Appendix 7. 
B.5.b. At what hospital or clinic will the treatment be given? Which facil ities of the 
hospital or clinic will be especially important for the proposed stud y? Will patients 
occupy regular hospital beds or clinical research center beds? Where will patients 
reside during the follow-up period? What special arrangements wil l be made for the 
comfort and consideration of the patients? Will the resear ch institution designate an 
ombudsman, patient care representative, or other individu al to help protect the rights 
and welfare of the patient? 
The study will be performed at the University of Iowa Medical Center in the Clinical 
Research Center. The University of Iowa Medical Center has all of the facilities required 
for the care of the patients; it is the largest teaching hospital in the country. Patients will be 
in single rooms in isolation. After there is no further evidence of live virus by two 
consecutive cultures, the patients will be discharged to their homes. We will try to offer 
entertainment (VCR, reading material, games), a stationary bicycle, and psychological 
Recombinant DNA Research, Volume 16 
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