Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases
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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.
KeywordsEnzyme Replacement Therapy Fabry Disease Lysosomal Storage Disorder Metachromatic Leukodystrophy Agalsidase Beta
TGC is the recipient of a CAPES scholarship; VLL and FQM are recipients of CNPq scholarships, UM and RG are recipient of CNPq PQ scholarships. This work was supported by FIPE-HCPA.
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