Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy-Reference-Cited by-同舟云学术

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Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy

Published:2010-07 Issue:1 Volume:30 Page:13-18
ISSN:1016-8478
Container-title:Molecules and Cells
language:en
Short-container-title:Mol Cells

Author:

Jung Sung-Chul,Park Eun-Sook,Choi Eun Nam,Kim Chi Hwa,Kim Su Jin,Jin Dong-Kyu

Publisher

Springer Science and Business Media LLC

Subject

Cell Biology,Molecular Biology,General Medicine

Link

http://link.springer.com/content/pdf/10.1007/s10059-010-0083-2.pdf

Reference32 articles.

1. Birkenmeier, E.H., Davisson, M.T., Beamer, W.G., Ganschow, R.E., Vogler, C.A., Gwynn, B., Lyford, K.A., Maltais, L.M., and Wawrzyniak, C.J. (1989). Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency. J. Clin. Invest. 83, 1258–1266.

2. Braun, S.E., Aronovich, E.L., Anderson, R.A., Crotty, P.L., McIvor, R.S., and Whitley, C.B. (1993). Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase. Proc. Natl. Acad. Sci. USA 90, 11830–11834.

3. Brooks, D.A., Kakavanos, R., and Hopwood, J.J. (2003). Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder. Trends Mol. Med. 9, 450–453.

4. Cardone, M., Polito, V.A., Pepe, S., Mann, L., D’Azzo, A., Auricchio, A., Ballabio, A., and Cosma, M.P. (2006). Correction of Hunter syndrome in the MPS II mouse model by AAV2/8-mediated gene delivery. Hum. Mol. Genet. 15, 1225–1236.

5. Clarke, L.A., Russell, C.S., Pownall, S., Warrington, C.L., Borowski, A., Dimmick, J.E., Toone, J., and Jirik, F.R. (1997). Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene. Hum. Mol. Genet. 6, 503–511.

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