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Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases

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Journal of Inherited Metabolic Disease

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.

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Acknowledgements

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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Authors and Affiliations

  1. Gene Therapy Center, Experimental Research Center, Hospital de Clínicas, Porto Alegre, RS, Brazil

    Ursula Matte, Valeska Lizzi Lagranha, Talita Giacomet de Carvalho, Fabiana Quoos Mayer & Roberto Giugliani

  2. Post-Graduation Program on Genetics and Molecular Biology, UFRGS, Porto Alegre, RS, Brazil

    Ursula Matte, Valeska Lizzi Lagranha, Talita Giacomet de Carvalho, Fabiana Quoos Mayer & Roberto Giugliani

  3. Post-Graduation Program on Child and Adolescent Health, UFRGS, Porto Alegre, RS, Brazil

    Ursula Matte & Roberto Giugliani

  4. Medical Genetics Service, Hospital de Clínicas, and Department of Genetics, UFRGS, Porto Alegre, RS, Brazil

    Roberto Giugliani

  5. Gene Therapy Center, Hospital de Clínicas de Porto Alegre, Rua Ramiro Barcelos, 2350, Porto Alegre, RS, 90035–903, Brazil

    Roberto Giugliani

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  1. Ursula Matte
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  2. Valeska Lizzi Lagranha
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  3. Talita Giacomet de Carvalho
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  4. Fabiana Quoos Mayer
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  5. Roberto Giugliani
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Correspondence to Roberto Giugliani.

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Communicated by: Gregory M. Pastores

Competing interest: None declared.

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Matte, U., Lagranha, V.L., de Carvalho, T.G. et al. Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases. J Inherit Metab Dis 34, 983–990 (2011). https://doi.org/10.1007/s10545-011-9350-4

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  • DOI: https://doi.org/10.1007/s10545-011-9350-4

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