AMELIORATION OF PROLIDASE DEFICIENCY IN FIBROBLASTS USING ADENOVIRUS-MEDIATED GENE-TRANSFER

Prolidase deficiency is an autosomal recessive inherited disease chara cterized clinically by frequent infections, mental retardation, and va rious skin lesions. Fundamental treatments for these manifestations ha ve not been established. We performed adenovirus-mediated gene transfe r of human prolidase cDNA into fibroblasts from patients with prolidas e deficiency. Infection with the adenovirus vector carrying human prol idase cDNA increased prolidase activity in fibroblasts up to approxima tely 7.5 times of that of normal control fibroblasts. This indicates t he feasibility of adenovirus-mediated gene therapy to treat patients w ith prolidase deficiency in the future.