Lymphocyte Gene Therapy for Mild Hunter Syndrome 
approximately 12 visits for apheresis and cell infusion for each patient. The entire study treating 4 
patients, each for 1 year, will extend over 18 - 24 months. 
Sedation may be required for follow-up studies (e.g., MRI and CT scans). 
This experimental treatment may provide benefit to the patient by producing a measurable 
improvement in cardiac and pulmonary function or general clinical status. The study also aims to 
evaluate the survival in the patient's blood of the infused IDS gene-modified T-lymphocytes and 
to determine whether autologous T-lymphocytes have prolonged survival in vivo, and have a 
selective growth advantage in vivo. 
4.5.2 Duration Of Treatment 
Each patient will receive 12 treatments given at monthly intervals over the course of 1 year. 
At the end of 1 year of treatment, a decision will be made regarding continuation or 
discontinuation of treatment based upon criteria established for this study and the availability of 
funds for continued treatment. 
4.6 Therapeutic Modifications 
4.6.1 During Apheresis 
In the extremely unlikely event that a subject has a hypotensive episode during apheresis, 
the transfer rate will be decreased or the procedure discontinued altogether based upon past 
experience of the technical personnel. Intravenous fluids (e.g., normal saline or albumin, 10 
ml /kg) will be administered if appropriate. 
4.6.2 During PBL Infusion 
If a subject has an adverse reaction during infusion (e.g., fever, rash or itching, tachycardia, 
tachypnea, hypotension or hypertension), the infusion will be slowed or discontinued altogether. 
Such episodes may also be treated with appropriate medications in standard doses (e.g., Tylenol 
10 - 15 mg/kg/dose; Benadryl 1- 2 mg/kg slow IV infusion; hydrocortisone 4-8 mg/kg dose IV). 
4.7 Study Parameters 
Clinical, biochemical and molecular genetic data will be collected as indicated on the 
attached schedule (Appendix A). 
4.8 Off-Study Criteria 
Patients will be taken "off study" (and treatment will be discontinued) for any one of the 
following reasons: 
4.8.1 Patient Request 
The patient will be taken "off treatment" if he requests to be taken "off treatment" for any 
specific or unspecified reason, or fails to appear for treatment. 
4.8.2 Inability To Monitor 
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