Publication: Gene transfer may be preventive but not curative for a lysosomal transport disorder
Gene transfer may be preventive but not curative for a lysosomal transport disorder
Date
Date
Date
Citations
Hippert, C., Dubois, G., Morin, C., Disson, O., Ibanes, S., Jacquet, C., Schwendener, R., Antignac, C., Kremer, E. J., & Kalatzis, V. (2008). Gene transfer may be preventive but not curative for a lysosomal transport disorder. Molecular Therapy, 16(8), 1372–1381. https://doi.org/10.1038/mt.2008.126
Abstract
Abstract
Abstract
Cystinosis belongs to a growing class of lysosomal storage disorders (LSDs) caused by defective transmembrane proteins. The causative CTNS gene encodes the lysosomal cystine transporter, cystinosin. Currently the aminothiol cysteamine is the only drug available for reducing cystine storage but this treatment has non-negligible side effects and administration constraints. In this study, for the first time, we report viral vector-mediated CTNS gene transfer and evaluate the feasibility of this strategy as a complementary treatment. Init
Additional indexing
Creators (Authors)
- Hippert, C
- Dubois, G
- Morin, C
- Disson, O
- Ibanes, S
- Jacquet, C
- Schwendener, R
- Antignac, C
- Kremer, E J
- Kalatzis, V
Volume
Volume
Volume
Number
Number
Number
Page range/Item number
Page range/Item number
Page range/Item number
Page end
Page end
Page end
Item Type
Item Type
Item Type
Language
Language
Language
Publication date
Publication date
Publication date
Date available
Date available
Date available
ISSN or e-ISSN
ISSN or e-ISSN
ISSN or e-ISSN
OA Status
OA Status
OA Status
Publisher DOI
Citations
Hippert, C., Dubois, G., Morin, C., Disson, O., Ibanes, S., Jacquet, C., Schwendener, R., Antignac, C., Kremer, E. J., & Kalatzis, V. (2008). Gene transfer may be preventive but not curative for a lysosomal transport disorder. Molecular Therapy, 16(8), 1372–1381. https://doi.org/10.1038/mt.2008.126
