Amelioration of prolidase deficiency in fibroblasts using adenovirus mediated gene transfer.
Prolidase deficiency is an autosomal recessive inherited disease characterized clinically by frequent infections, mental retardation, and various skin lesions. Fundamental treatments for these manifestations have not been established. We performed adenovirus-mediated gene transfer of human prolidase cDNA into fibroblasts from patients with prolidase deficiency. Infection with the adenovirus vector carrying human prolidase cDNA increased prolidase activity in fibroblasts up to approximately 7.5 times of that of normal control fibroblasts. This indicates the feasibility of adenovirus-mediated gene therapy to treat patients with prolidase deficiency in the future.
Ikeda
K
K
Department of Inherited Metabolic Disease, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawahigashi, Kodaira, Tokyo 187, Japan.
Tohyama
J
J
Tsujino
S
S
Sato
K
K
Oono
T
T
Arata
J
J
Endo
F
F
Sakuragawa
N
N
D000256:Adenoviridae / Q000235:genetics*; D002478:Cells, Cultured; D018076:DNA, Complementary / Q000235:genetics; D004150:Dipeptidases / Q000172:deficiency* / Q000235:genetics* / Q000378:metabolism; D005260:Female; D005347:Fibroblasts / Q000201:enzymology / Q000378:metabolism; D018014:Gene Transfer Techniques; D005808:Genes, Recessive; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008661:Metabolism, Inborn Errors / Q000201:enzymology / Q000628:therapy*; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism; D012867:Skin / Q000166:cytology; D012871:Skin Diseases / Q000201:enzymology / Q000235:genetics
D018076:DNA, Complementary; D012333:RNA, Messenger; D004150:Dipeptidases; C022449:proline dipeptidase