Microencapsulation—A Novel Gene Therapy for Lysosomal Storage Diseases

  • Colin J. D. Ross
  • Patricia L. Chang

Abstract

Over 30 different lysosomal storage diseases, each associated with deficiency of a specific lysosomal enzyme, have been described in man (Gieselmann 1995). Because of the ubiquitous presence of lysosomes in almost all cell types, deficiencies of these enzymes cause multisystem anomalies, often with catastrophic consequences, and frequently result in early death. Although the incidence of individual enzyme deficiency is rare, lysosomal storage diseases collectively occur in 1 in∼10,000 births. In spite of such significant prevalence and devastating consequences, there is no cure or even definitive treatment for most of these diseases. Until recently, bone marrow transplantation has been the only experimental treatment offered with some degree of success (Krivit et al 1990, Hoogerbrugge et al 1995). However, this procedure carries high rates of morbidity (graft-versus-host disease) and mortality (10% in matched and 20–25% in unmatched donors). Furthermore, even if the risks are acceptable, most patients (70%) cannot find compatible donors (Parkman 1986). In the majority of the affected families the only medical interventions available are palliative care until death occurs, and prevention of recurrence through prenatal diagnosis. An alternative treatment available to a subtype of one form of lysosomal storage disease is enzyme replacement. For the non-neuropathic form of Gaucher’s disease, administration of glucocerebrosidase, the deficient enzyme in question, has resulted in definite clinical imrovements. This therapy is now an accepted form of treatment in the U.S., but only for those who can afford it. Under the Orphan Drug Act, with no competitive markets, the annual cost for the enzyme Ceredase alone is $380,000/70 kg (FDA recommendation, 1991). Because of the risks and the dificulty in locating donors for bone marrow transplant, and the high cost for either bone marrow transplantation (>$ 100,000) or enzyme replacement treatment, more cost-effective therapies for the lysosomal storage diseases need to be developed. Since the genes for many lysosomal enzymes have been cloned, somatic gene therapy for lysosomal storage diseases may offer the ultimate solution to this serious healthcare problem (Beutler 1993).

Keywords

Alginate Stein Encapsulation Allo Univer 

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© Springer Science+Business Media New York 1999

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  • Colin J. D. Ross
  • Patricia L. Chang

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