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Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases
Author(s) -
Matte Ursula,
Lagranha Valeska Lizzi,
Carvalho Talita Giacomet,
Mayer Fabiana Quoos,
Giugliani Roberto
Publication year - 2011
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-011-9350-4
Subject(s) - lysosomal storage disorders , substrate reduction therapy , enzyme , enzyme replacement therapy , lysosome , mannose , cell , glucocerebrosidase , lysosomal storage disease , mannose 6 phosphate receptor , receptor , biology , microbiology and biotechnology , biochemistry , medicine , disease
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.