Preface 
This document is submitted in response to the memorandum of Dr. William J. 
Gartland. dated February 25. 1986. which requested that investigators supply 
preclinical data to the Human Gene Therapy Subcommittee of the NIH RAC for 
"information purposes and part of an education process for the Subcommittee and 
submitters" and not as "part of the formal review and approval process". We 
emphasize that our submission is not a clinical proposal nor do we request any 
form of approval for a clinical protocol at this time. Furthermore, this 
document is not a final submission, but only an initial communication. Some 
issues are still being debated within and among our own laboratories: many 
experiments are ongoing. Therefore, we anticipate that this document will be 
added to, and modified, in light of new results as well as in response to 
questions from the Subcommittee or others. 
We address each of the questions raised in the "Points to Consider in the 
Design and Submission of Human Somatic Cell Gene Therapy Protocols" (each 
"Point" is typed single-spaced in bold print) with respect to gene transfer 
using a retroviral vector for correcting inherited ADA deficiency. In 
attempting to answer the queries, the following three questions have emerged. 
We feel that they are of fundamental importance and will require careful 
consideration by the Subcommittee prior to the time that a clinical protocol be 
formally considered. 
[4] 
Recombinant DNA Research, Volume 12 
