O. Sabate et al., TRANSPLANTATION TO THE RAT-BRAIN OF HUMAN NEURAL PROGENITORS THAT WERE GENETICALLY-MODIFIED USING ADENOVIRUSES, Nature genetics, 9(3), 1995, pp. 256-260
Transplantations for neurological disorders are limited by the supply
of human fetal tissue. To generate larger numbers of cells of appropri
ate phenotype, we investigated whether human neural progenitors expand
ed in vitro could be modified with recombinant adenoviruses. Strong ex
pression of beta-galactosidase was obtained in vitro. Two or three wee
ks after transplantation of engineered cells to the rat brain, we obse
rved a small percentage of surviving neuroblasts strongly expressing b
eta-galactosidase in four out of 13 rats. Thus human precursor cells t
hat have been genetically modified using adenoviruses are a promising
tool for ex vivo gene therapy of neurodegenerative diseases.