National Heart, Lung, and Blood Institute
CENTER FOR FETAL MONKEY GENE TRANSFER FOR
HEART, LUNG, AND BLOOD DISEASES
 
Home
About the Center
Call for Letters of Intent
Services Offered to Grantees
Funded Projects
Related Links
Contact

Annual Gene Therapy Symposium
California National Primate Research Center
Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases: A NHLBI Resource for the Gene Therapy Community
National Heart, Lung, and Blood Institute
NHLBI Scientific Resources
American Society of Gene and Cell Therapy
Gene Therapy Resource Program
UC Davis
One UC Davis
 
Funded Projects
 
   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

   2008-2009

  • Early AAV-mediated gene therapy for genetic disorders: A pilot study in nonhuman primates
  • Human stem cell based therapy for the heart

   2009-2010

  • Systemic and cardiac gene transfer with novel AAV vectors
  • Study of efficiency, stability, and safety of AAV8-mediated, liver directed gene transfer in neonatal nonhuman primates
  • Administration of cKIT blocking antibody to juvenile rhesus monkeys and effects on hematopoiesis

   2010-2011

  • Administration of c-kit blocking antibody to juvenile rhesus monkeys and effects on engraftment of lentivirus-transduced autologous marrow
  • Evaluation of the safety and expression of Hexon PEGylated Helper-dependent adenoviral vector expressing human FVIII in non-human primates
  • Translational primate studies to assess efficacy and safety of autologous transplantation and chemoselection
  • Developmental modulation of fibrosis in fetal monkey lung

   2011-2012

  • In Vivo Transduction by IV Injection of a Lentiviral Vector Expressing ADA: Pegylated VSV Lentiviral Vectors for Decreased Immune Response
  • Studies on Repeat Administration of AAV Vector for Cardiac and Respiratory Myopathies
  • Examination of the Immune Response to Transgene-Encoded Proteins with Expression Beginning in the Early Neonatal Period

   2012-2013

  • A Model for Bronchopulmonary Dysplasia in Extremely Preterm Infants
  • Costimulation Blockade to Prevent Stem Cell Graft Rejection In Vivo

   2013-2014

  • Induced Pluripotent Stem Cells (iPSC) in the Understanding and Treatment of Heart Disease
  • Neonatal Tolerance Induction to Facilitate Subsequent AAV Directed Gene Therapy
 



 

 
Home  |  About the Center  |  Call for Letters of Intent  |  Services Offered to Grantees
Funded Projects  |  Related Links  |  Contact