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Type: Journal article
Title: Directed differentiation and characterization of genetyically modified embryonic stem cells for therapy
Author: Lau, A.
Hemsley, K.
Meedeniya, A.
Robinson, A.
Hopwood, J.
Citation: Methods in Molecular Biology, 2006; 329:471-484
Publisher: Humana Press
Issue Date: 2006
ISSN: 1064-3745
Statement of
Adeline A. Lau, Kim M. Hemsley, Adrian Meedeniya, Aaron J. Robinson and John J. Hopwood
Abstract: Lysosomal storage disorders are rare, inherited diseases caused by a deficiency of a specific, lysosomal enzyme. In the case of mucopolysaccharidosis type IIIA, a lack of active sulfamidase enzyme results in heparan sulfate accumulation, severe and progressive neurological deficits, and usually premature death. Embryonic stem cells can be genetically modified to overexpress lysosomal enzymes, providing a renewable reservoir of cells that can be readily expanded in culture. Screening clonal lines of embryonic stem cells for desirable properties such as high levels and maintenance of enzyme activity throughout terminal differentiation to neural phenotypes theoretically provides a reproducible population of cells that can be fully characterized in vitro before implantation within the central nervous system in animal models of lysosomal storage disorders.
Keywords: Clonal; differentiation; embryonic stem cells; in vitro; lysosomal enzyme; lysosomal storage; therapy; transgenic
Rights: © Humana Press Inc., Totowa, NJ
RMID: 0020063042
DOI: 10.1385/1-59745-037-5:471
Appears in Collections:Paediatrics publications

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