TABLE OF CONTENTS 
1.0 Precis of Amendment 4 
2.0 Objectives 5 
3.0 Scientific Rationale and Background 5 
3.1 Rationale 
3.2 Enrichment for CD34+ Cells 
3.3 Ex vivo Maintenance and Expansion of Hematopoietic 
Progenitors 
3.4 Feasibility of Gene Transfer to Hematopoietic 
Progenitors and Stem Cells 
3.5 Feasibility of Gene Transfer to Tumor Cells 
3.6 Safety of the Transduction Procedure 
4.0 Patient Eligibility 16 
5.0 Treatment Plan 17 
6.0 On-Study Evaluation 18 
7.0 Hazards and Discomforts 20 
8.0 Toxicity Monitoring and Reporting 20 
9.0 Criteria for Removal from Amendment 21 
10.0 Response Criteria and Impact of Outcome 21 
11.0 References 24 
12.0 Appendices 30 
A-Schema of Genetic Marking Amendment 31 
B-CD34 Selection and Retroviral 
Transduction Procedures 32 
C-In vitro Analysis of CD34-Enriched 
and Transduced Cells 39 
D-Summary of Pre-Clinical Gene Transfer Data 40 
E-Summary Diagram: Retroviral Gene Transfer 44 
F-LNL6 and GIN. 40 Vectors: Diagrams of 
Structure and Description of Differences 45 
G-Certif icate of Analysis for Retroviral 
Supernatants Obtained from Genetic 
Therapy Inc.(GTI) 1 48 
H-Certif icate of Analysis for Transduced Cells 50 
I-Letter to FDA re: Lymphoma in Gene Transfer 
Monkeys 51 
13.0 Informed Consent Documents 
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Recombinant DNA Research, Volume 16 
