title: Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases.
abstract: Lysosomal storage disorders (LSD) are monogenic diseases caused by the deficiency of different lysosomal enzymes that degrade complex substrates such as glycosaminoglycans, sphingolipids, and others. As a consequence there is multisystemic storage of these substrates. Most treatments for these disorders are based in the fact that most of these enzymes are soluble and can be internalized by adjacent cells via mannose-6-phosphate receptor. In that sense, these disorders are good candidates to be treated by somatic gene therapy based on cell microencapsulation. Here, we review the existing data about this approach focused on the LSD treatments, the advantages and limitations faced by these studies.
authors: Gene Therapy Center, Experimental Research Center, Hospital de Clínicas, Porto Alegre, RS, Brazil.
authors: Ursula
authors: U
authors: Matte
authors: Valeska Lizzi
authors: VL
authors: Lagranha
authors: Talita Giacomet
authors: TG
authors: de Carvalho
authors: Fabiana Quoos
authors: FQ
authors: Mayer
authors: Roberto
authors: R
authors: Giugliani
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