title: Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.
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.
authors.affiliation: Gene Therapy Laboratory, Department of Pediatrics, University of Padova, Italy.
authors.forename: Rosella
authors.initials: R
authors.lastname: Tomanin
authors.forename: Alessandra
authors.initials: A
authors.lastname: Zanetti
authors.forename: Eva
authors.initials: E
authors.lastname: Zaccariotto
authors.forename: Francesca
authors.initials: F
authors.lastname: D'Avanzo
authors.forename: Cinzia M
authors.initials: CM
authors.lastname: Bellettato
authors.forename: Maurizio
authors.initials: M
authors.lastname: Scarpa
mesh_terms: D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy*