Journal
ACTA PAEDIATRICA
Volume 101, Issue 7, Pages 692-701Publisher
WILEY
DOI: 10.1111/j.1651-2227.2012.02674.x
Keywords
Gene therapy; Lysosomal storage disorders; Mendelian diseases
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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.
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