APPENDIX C 
TABLE OF CONTENTS 
Protocol #838.0 Protocol 
1. Title of Protocol 
2. Introduction 
3. Background 
A. Pathophysiology of Gaucher’s disease 
B. Retrovirus-mediated transduction of the human glucocerebrosidase cDNA into murine 
long-term repopulating marrow cells 
C. Retrovirus-mediated transduction of the neomycinphosphotransferase (neo) and the 
human adenosine deaminase (ADA) gene into canine long-term repopulating marrow 
cells 
D. Retrovirus-mediated neo gene transduction into canine long-term repopulating 
peripheral blood cells 
E. Retrovirus-mediated transduction of the human glucocerebrosidase gene into canine 
long-term repopulating hematopoietic cells 
F. Retrovirus-mediated neo gene transduction into human G-CSF mobilized peripheral 
blood-derived CFU-GM 
G. Retrovirus-mediated transduction of the human glucocerebrosidase gene into human 
hematopoietic progenitor cells 
H. Ex vivo neo gene transduction into human marrow cells followed by autologous marrow 
transplantation 
I. Engraftment of hematopoietic repopulating cells in nonmyeloablated recipients 
J. Safety issues of retrovirus-mediated gene transduction 
4. Objectives 
5. Patient Selection 
A. Inclusions 
B. Exclusions 
6. Evaluation and counseling of patient 
7. Protocol registration 
8. Plan of treatment 
A. Treatment diagram 
B. G-CSF administration 
C. Peripheral blood cell collection 
D. Separation of CD34 + peripheral blood cells 
E. Transduction of CD34+ peripheral blood cells 
F. Infusion of transduced CD34+ peripheral blood cells 
G. Frequency of infusion of transduced CD34 + peripheral blood cells 
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