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.
- Baruch L, Benny O, Gilert A, Ukobnik M, Ben Itzhak O, Machluf M (2009) Alginate-PLL cell encapsulation system Co-entrapping PLGA-microspheres for the continuous release of anti-inflammatory drugs. Biomed Microdevices 11:1103–1113Google Scholar
- Blasi P, Giovagnoli S, Schoubben A, Ricci M, Rossi C, Luca G, Basta G, Calafiore R (2006) Preparation and in vitro and in vivo characterization of composite microcapsules for cell encapsulation. Int J Pharm. 31;324(1):27–36.Google Scholar
- Chang PL (1999) Encapsulation for somatic gene therapy. Ann N Y Acad Sci 18:(875):146–158. Mater ResGoogle Scholar
- Lowry RB, Renwick DH (1971) Relative frequency of the Hurler and Hunter syndromes. N Engl J Med 28;284(4):221–2.Google Scholar
- Mabe-Santana P (2006) La enfermedad de Krabbe y la leucodistrofia metacromática. In: Sanjurjo P, Baldellou A (eds.) Diagnóstico y tratamiento de las enfermedades metabólicas hereditárias: Ergon, 639–650Google Scholar
- Martin RA (2007) Mucopolysaccharidosis Type II. In: Pagon RA, Bird TC, Dolan CR, Stephens K (eds.) GeneReviews [Internet]. Seattle (WA): University of Washington, SeattleGoogle Scholar
- National MPS Society, viewed August 14 2010, http://www.mpssociety.org
- Neufeld EF, Muenzer J (2001) The mucopolysaccharidoses. In: Scriver C, Beaudet A, Sly W et al. (eds) The metabolic and molecular bases of inherited disease. McGraw Hill, New York, pp 3421–3452Google Scholar