Phenotypic correction of dwarfism by constitutive expression of growth hormone.

GH is normally secreted in a pulsatile fashion. When GH is deficient, dwarfism is the result in both rodents and humans. An adenoviral vector containing the rat GH complementary DNA was used to induce constitutive GH expression in hepatocytes of GH-deficient lit/lit mice. Elevated serum GH increased circulating insulin-like growth factor I concentrations, corrected the growth deficiency, and normalized body composition. The results indicate that correction of the dwarf phenotype can be achieved by constitutive expression of GH at an ectopic site by gene transfer.