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4. Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

6. Safety profile after prolonged C3 inhibition

8. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates

10. Effects of Vector Backbone and Pseudotype on Lentiviral Vector-mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys

12. Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

16. The Biomedical Resource Ontology (BRO) to enable resource discovery in clinical and translational research

23. Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector

24. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): Safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery

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