UNIVERSITY OF CALIFORNIA, SAN DIEGO 
BERKELEY • DAVIS • IRVINE • LOS ANGELES • RIVERSIDE • SAN DIEGO • SAN FRANCISCO 
SANTA BARBARA • SANTA CRUZ 
DEPARTMENT OF NEUROSCIENCES M-008 LA JOLLA, CALIFORNIA 92093 
SCHOOL OF MEDICINE 
October 5, 1984 
Director 
Office of Recombinant DNA Activity 
Building 31, Room 3B10 
National Institutes of Health 
Bethesda, Maryland 20205 
Dear Sir: 
I am writing in response to the letter of Mr. Jeremy Rifkin in the Federal Register 
of September 20, 1984, in which he wishes to outlaw gene transfer experiments in 
animals. If such were done it would make impossible experiments which need to be 
performed to determine the feasibility of gene transfer experiments in humans. My 
own particular field is the genetics of inherited neurological disease. In 1969 I 
discovered the basic genetic defect in Tay-Sachs disease. We are currently working 
out techniques to see if gene therapy could be used to cure patients with Tay- 
Sachs disease and related diseases by gene transfer. Such experiments will involve 
the isolation of the normal gene which is involved in these diseases, the insertion 
of such genes into appropriate vectors and studies in cultured cell systems and 
whole animals to determine the effectiveness of such gene transfer on the 
correction of the metabolic defect. Several animal models for ganglioside storage 
diseases are currently available in cats, dogs and mice and these are ideal subjects 
to determine the effectiveness of gene therapy as a prelude to any studies in 
humans. 
If such research is outlawed it would make impossible the long term carefully 
planned and detailed studies required in order to maximize conditions for human 
therapeutic studies. There are thousands of families throughout the world who have 
had a genetically defective child and are anxiously awaiting the results of this 
research with high hope and great expectation. I wish therefore, to strongly speak 
against the position taken outlawing such experiments. 
Sincerely yours, 
Professor of Neurosciences 
JOB /so 
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