a. Limiting dilution quantitation and DNA PCR to assess transduced cell survival in 
vivo. PCR primers will be chosen to acheive amplification specific for the 
ribozyme-containing vector and control vector. The longevity of the 
transduced cells, the primary experimental variable to be monitored in this 
trial, is difficult to prospectively estimate. Nevertheless, we note that in the ADA 
trial a large number of gene-corrected T cells survived in the first patient for 
over six months after cell infusions were stopped, a result that confounded pre- 
trial concerns that cells would have a severely short lifespan (2). 
b. RNA PCR to assess ribozyme expression in vivo. 
c. CTL assay. 
d. Virus isolation. 
e. Phenotype of transduced cells in vivo. 
3. CD4 count 
4. p24 antigen level. 
5. Complete blood counts and standard chemistries (SMA-20). 
TABLE 1 
Week: 1 
2 
3 4 
8 1 2 
1 6 
2 0 
2 4 
3 2 
5 2 
Test 
*1 . X 
X 
X X 
X X 
X 
X 
X 
X 
X 
2 - a . X 
X 
X 
X 
X 
X 
X 
2 - b . X 
X 
X 
X 
X 
X 
X 
o 
■ 
CM 
X 
X 
M 
• 
CL 
X 
X 
2 - e . X 
X 
X 
3 . X 
X 
X 
X 
4 . X 
X 
X 
X 
5 . X 
X 
X X 
X X 
X 
X 
X 
X 
X 
'Numbers refer to designations in immediately preceding section 
[3941 
Recombinant DNA Research, Volume 18 
