Genzyme Corporation Ad2/CFTR-2 Gene Transfer Protocol: Single 
One Kendall Square, Cambridge, MA 02139 Aerosol Administration to the Lung of CF Patients 
PROTOCOL FLOW CHART - This chart illustrates our proposed aerosolization protocol as well as the 
concurrent lobar protocol which is shown in the shaded boxes. 
Protocol/Dose 
1994 1995 
IU 1 Nov Dec Jan Feb Mar Apr May Jun Jul Aug Sep Oct 
Lobar/1 
8x10® PE •: ,FE;| 
Aerosol/1 
8x10® PE n=2 FE 
Lobar/2 
-V:. 1 
2.5x10? PE n=3 FE 
Aerosol/2 
2.5 xIO 7 PE n=2 FE 
Lobar/3 
8x10 7 PE n=3 FE 
Aerosol/3 
8x10 7 PE n=2 FE 
w\i. 
Aerosol/4 
25x10® , »*■ l :;-PE:iv,n=3^,TEv; 
2.5x10® PE n=2 FE 
Lobar/5 
8x10® PE n=3 FE 
Aerosol/5 
8x10® PE n=2 FE 
Lobar/6 . 
2.5 xIO 9 PE n=3 FE 
IS i & * M , £2 j 
2.5x10 9 PE n=2 FE 
Aerosol/6 
Aerosoi/7 
8x10® PE n=3 FE 
8x10 9 PE n=2 FE 
Lobar/8 
25x10 10 PE n=3 FE 
Aerosol/8 
2.5 x 10 10 PE n=2 FE 
PE = Pre-treatment evaluations FE = Post-treatment follow up evaluations 
"•This assumes the clinical lot has a particle to IU ratio of 100/1 . 
Recombinant DNA Research, Volume 20 
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