TABLE OF CONTENTS 
Page 
1.0 CHECKLIST FOR ELIGIBILITY 3 
2.0 OBJECTIVES 3 
3.0 BACKGROUND AND RATIONALE 3 
4.0 STUDY OUTLINE 6 
5.0 STUDY DESIGN 7 
6.0 PATIENT ELIGIBILITY 7 
7.0 TREATMENT PLAN 8 
8.0 PATIENT EVALUATION 8 
9.0 EVALUATION DURING STUDY 9 
10.0 STATISTICAL CONSIDERATION 9 
11.0. RECORDS TO BE KEPT 12 
12.0. REPORTING REQUIREMENTS 12 
13.0 INFORMED CONSENT 13 
14.0 REFERENCES 14 
PARENTS SUMMARY STATEMENT 16 
APPENDIX A: Pre-Clinical Data on Selection and Ex Vivo Expansion of CD34 Cells 
APPENDIX B: Marrow Preparation 
APPENDIX C: Transduction of Marrow 
APPENDIX D: Incubation with Growth Factors 
APPENDIX E; Dartmouth Criteria for Engraftment 
APPENDIX F: Marrow Reinfusion 
APPENDIX G: NIH Toxicity Criteria 
Recombinant DNA Research, Volume 19 
[489] 
