9 
Dr. Gottesman said the purpose of human gene therapy does not differ from the 
purposes of other therapies, and the consequences of human gene therapy need 
not differ if it is applied correctly. She suggested the words "in terms of 
consequences" be substituted for the words "in clinical terms." The working 
group accepted Dr. Gottesman' s proposed language. Mr. Capron volunteered to 
attempt to draft langauge for this section of the document. 
Dr. Rich suggested the document should include a statement on the distinction 
between vertical and horizontal transmission of genes. 
Dr. Rich said the working group apparently wishes to construct two different 
documents. The first document would explain human gene therapy in nontechnical 
terms and ccrrmunicate to the public that the working group is considering these 
issues. The other document would be a technical document specifying the infor- 
mation the working group would like to review. Dr. Rich said the working 
group needs both documents; he suggested two documents be drafted and appended 
one to the other. 
Dr. Grobstein also questioned whether the working group could deal with both 
of these issues simultaneously in the same document. Dr. Walters said the 
points to consider document is directed to two different audiences, the public 
and the investigator. He felt the introduction to the document could be directed 
to both of these audiences. 
Ms. Witherby supported Dr. Rich's proposal to prepare two documents. She felt 
all the social issues will come to life when the first protocol is submitted 
for review; the working group should have at that time a document to explain 
gene therapy in nontechnical terms and discuss the social issues. 
Dr. Anderson said the working group should cite the history of introducing 
genetic material into humans. The first successful bone marrow transplant was 
performed in 1968. A young male patient was treated for ADA deficiency; his 
T-cells wore completely replaced by T-cells from a female donor. No one questions 
the male identity of this patient even though he carries genes from the female 
donor. Dr. Anderson said the changes which would be introduced into the patient 
through human gene therapy are smaller than the addition of genetic material 
which occurs with bone marrow transplants. 
Dr. Grobstein said bone marrow transplants and human gene therapy replacement 
of a defective gene are not fundamentally different yet the public may perceive 
these two procedures differently. The working group should be prepared to ad- 
dress this difference in perception. 
Dr. Walters again drew the attention of the working group to Dr. Grobstein' s 
proposed draft language (Attachment V). 
Dr. Temin said question number two in Dr. Grobstein' s proposed language 
(Attachment V) is not a social issue. That question asks how good is the 
evidence that the proposed somatic cell gene therapy will not inadvertently 
affect reproductive cells. 
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