2001-2002

• Pre and postnatal AAV delivery in a primate model
• In vivo tropism of novel lentiviral envelope pseudotypes in fetal primates
• Gene transfer into blood stem cells of the fetal monkey
• Expression of factor IX and VIII genes following transduction of fetal tissue by lentiviral vectors

   2002-2003

• Engraftment and tolerance induction in utero in fetal rhesus monkeys
• Expression of SIN lentiviral vectors carrying the ß-globin expression cassette in nonhuman primates
• Expression of acid alpha-glucosidase in primate diaphragm after direct in utero delivery of recombinant AAV type 1
• Recombinant human parvovirus vector-mediated gene transfer in fetal monkeys

   2003-2004

• Fetal gene therapy using a non-viral Sleeping Beauty transposon delivery system
• Monkey gene transfer in HSCs by using a site-specific integrase
• Stem cell engraftment in the fetal lung
• In utero coagulation factor gene transfer
• TGF-ß1 viral transgene misexpression in monkey lung

   2004-2005

• Systemic gene transfer in late gestation or in newborn nonhuman primates - Implications for the treatment of metabolic disease in infancy
• Gene transfer for hemophilia in nonhuman primates using viral and nonviral vectors
• Induction of tolerance to a foreign protein in the monkey HSCT model
• Direct injection of recombinant lentivector into neonatal primates for correction of lysosomal storage disorders
• Engraftment of endothelial stem cells in the fetal monkey
• Imaging stem cell selection during fetal and neonatal development

   2005-2006

• Tolerance induction in fetal rhesus monkeys
• Novel approaches for engraftment after hematopoietic stem cell transplantation
• In vivo transduction by intravenous injection of lentiviral vector expressing human ADA; Potential novel therapy for ADA enzyme replacement
• Neonatal gene therapy for hemophilia B in rhesus macaques
• Evaluation of chimeric AAV vector for heart and liver tropic delivery
• Pre-clinical in utero and newborn gene transfer in nonhuman primates for hemophilia using AAV vectors

   2006-2007

• In vivo transduction by intravenous injection of a lentiviral vector expressing Adenosine Deaminase (ADA):  A novel therapy for ADA-deficient SCID
• Non-invasive assessment of gene transfer in newborn nonhuman primates-Implications for treatment of neuromuscular disorders in infancy
• Analysis of mesenchymal stem cell trafficking to bone and differentiation to osteoblasts in utero
• IL-7 gene therapy of mesenchymal stem cells and post-transplant immune reconstitution

   2007-2008

• Early AAV-mediated gene therapy for genetic disorders: A pilot study in nonhuman primates
• Neonatal gene therapy for hemophilia A in rhesus macaques
• Induction of tolerance to transgene products by immunoablative conditioning prior to gene transfer/bone marrow transplantation