Gene Transfer Approaches for Hemophilia A

Project Aims1.

To evaluate the therapeutic potential of two forms of genetically-modified autologous stem cells -

a)  Adult endothelial progenitor cellsa)
b)  Induced pluripotent stem cells

for the treatment of Hemophilia A.



Ex Vivo Gene Transfer


An Endothelial Progenitor Cell
BOEC (Blood Outgrowth Endothelial Cell)



Omental Biopsies of Implanted Endothelial Progenitor Cells


- 3 months after implantation into a hemophilic dog
- Red-staining cells are expressing Factor VIII

SPECIFIC AREAS OF RESEARCH ACTIVITY
Physiology and
Pathobiology of
von Willebrand Factor
The
Immunology
of Factor VIII
Gene Transfer
Approaches
for Hemophilia A