Recombinant DNA Advisory Committee - 5/30-31/91 
BACKGROUND INFORMATION ON THE REGISTRY OF GENE TRANSFER PATIENTS 
ENTITLED, THE GENE TRANSFER PATIENT AND PROVIDER NETWORK (GENETRA- 
NET): 
Dr. Ledley said this proposal was a corollary to his proposal which he presented yesterday 
which he had developed as a result of his clinical training in the field of phenylketonuria 
(PKU). He described the normal clinical course of PKU, noting that one of the problems was 
the ability, once the problem was corrected, to track patients so that doctors could continue 
to assess their long-term outcome as well as to ensure that the patients, some of whom were 
treated at a very early age, knew that they had a congenital genetic disease which and that 
they continue to carry this genotype. 
He said he felt the same type of network needed to be set up for gene transfer patients 
patterned after the "Register of Selected Inherited Metabolic Diseases (RSIMD)." He said 
this registry was used as a model because it already had been operating for a number of years 
and had in place the computer resources, personnel resources and other mechanical things to 
do it in a way that was both ethical and feasible. He said the same company that had 
collaborated on developing the RSIMD, Mize Information Enterprises, of Dallas, Texas, was 
being proposed to collaborate on this registry. 
Dr. Ledley pointed to some of the things this registry could help to track. He said the 
following are issues dealing with the use of the viral vector: 
1. Viral disease as a result of a defective vector or naturally recombinant viruses; 
2. Insertional mutagenesis, leading either to malignancies, cell damage, or perhaps 
premature senescence of cells; 
3. Contamination of vectors with other viruses; 
4. Long-term immune response against either the recombinant gene products or 
retroviral vectors; 
5. The issue of possible inheritable genetic damage; 
6. Psychosocial pathology affecting either self-image, family, or social structures; 
and, 
7. The stability and duration of functions that are put into the patient. 
He noted that one good reason for having such a registry is to find adverse reactions and to 
make inferences about what this means relative to clinical practice, which requires large 
numbers of patients to be able to determine. He said this registry would be set up with the 
following goals: 
Recombinant DNA Research, Volume 14 
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