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- Title
Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.
- Authors
Tomanin, Rosella; Zanetti, Alessandra; Zaccariotto, Eva; D'Avanzo, Francesca; Bellettato, Cinzia M; Scarpa, Maurizio
- Abstract
This review describes the different gene therapy technologies applied to approach lysosomal storage disorders, monogenic conditions, with known genetic and biochemical defects, for many of which animal models are available. Both viral and nonviral procedures are described, underlying the specific needs that the treatment of genetic disorders requires. Conclusions: Lysosomal storage disorders represent a good model of study of gene therapeutic procedures that are, or could be, relevant to the treatment of several other mendelian diseases.
- Subjects
THERAPEUTICS; GENETIC engineering; MEDICAL genetics; COMPARATIVE pathology; GENETIC disorders; ANIMAL disease models; GENE therapy
- Publication
Acta Paediatrica, 2012, Vol 101, Issue 7, p692
- ISSN
0803-5253
- Publication type
Article
- DOI
10.1111/j.1651-2227.2012.02674.x