Organ transplantation is now the definitive therapy for many forms of end-o
rgan disease, but chronic allograft rejection, the side effects of chronic
immunosuppressive therapy and the severe donor organ shortage continue to l
imit its success. Gene therapy has the potential to prevent graft rejection
by manipulating the immune response in the microenvironment of the graft o
r by facilitating the induction of tolerance, Genetic manipulation of stem
cells to create transgenic and/or knockout animals that could serve as orga
n or cell donors could be combined with gene therapy approaches to overcome
the problem of limited allogeneic donor organ supply.