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Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria

Title
Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria
Authors
Park E.-S.Oh H.-J.Kruger W.D.Jung S.-C.Lee J.-S.
Ewha Authors
정성철
SCOPUS Author ID
정성철scopus
Issue Date
2006
Journal Title
Experimental and Molecular Medicine
ISSN
1226-3613JCR Link
Citation
vol. 38, no. 6, pp. 652 - 661
Indexed
SCI; SCIE; SCOPUS; KCI WOS scopus
Abstract
Homocystinuria is a metabolic disorder caused by a deficiency of cystathionine β-synthase (CBS). The major clinical symptoms of this disease are mental retardation, lens dislocation, vascular disease with life-threatening thromboembolisms, and skeletal deformities. The major treatments for CBS deficiency include pharmacologic doses of pyridoxine or dietary restriction of methionine. There is currently no effective long-term treatment to lower the elevated plasma levels of homocysteine. However, gene therapy could be an effective novel approach for the treatment of homocystinuria. A recombinant adeno-associated virus vector carrying human CBS cDNA (rAAV-hCBS) was constructed and administered to CBS-/- mice by intramuscular (IM) and intraperitoneal (IP) injections. Serum homocysteine concentrations significantly decreased in treated mice compared with age-matched controls two weeks after treatment. The treated CBS-/- mice had life spans 3-7 days longer compared with untreated CBS-/- mice. In CBS-/- mice treated with rAAV-hCBS via IP injection, the vector was detected in all organs examined including liver, spleen, and kidney, and CBS gene expression was observed by immunohistochemical staining in the liver. These results indicate the efficacy of gene delivery and demonstrate the possibility of gene therapy mediated by AAV gene transfer in this mouse model of homocystinuria.
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의학전문대학원 > 의학과 > Journal papers
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