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Cell Encapsulation Technology and Therapeutics pp 330–342Cite as

Birkhäuser

Microencapsulation—A Novel Gene Therapy for Lysosomal Storage Diseases

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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).

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References

  • Aebischer P, Wahlberg L, Tresco PA, and Winn SR. 1991a. Macroencapsulation of dopamine-secreting cells by coextrusion with an organic polymer solution. Biomaterials. 12: 50–56.

    Article  PubMed  CAS  Google Scholar 

  • Aebischer P, Tresco PA, Sagen J, and Winn SR. 1991b. Transplantation of microencapsulated bovine chromaffin cells reduces lesion-induced rotational asymmetry in rats. Brain Res. 560: 43–49.

    Article  PubMed  CAS  Google Scholar 

  • Al-Hendy A, Hortelano G, Tannenbaum GS, and Chang PL. 1995. Correction of the growth defect in dwarf mice with non-autologous micro-encapsulated myoblasts— an alternate approach to somatic gene therapy. Hum. GeneTher. 6: 165–175.

    CAS  Google Scholar 

  • Anderson WF. 1995. Gene therapy. Sci. Am. 273:124–128.

    PubMed  CAS  Google Scholar 

  • Awrey D, Tse M, Hortelano G, and Chang PL. 1995. Permeability of alginate microcapsules to secretory recombinant gene products. Manuscript submitted

    Google Scholar 

  • Bastedo L, Sands MS, Hortelano B, Al-Hendy A, and Chang PL. 1994. Partial correction of murine mucopolysaccaridosis VII with micro-encapsulated non-autologous recombinant fibroblasts. Am. J. Hum. Genet. 55:A211.

    Google Scholar 

  • Bastedo L, Sands MS, Lambert DT, Pisa MA, Birkenmeier E, and Chang P. 1994. Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII. J. Clin. Invest. 94:1180–1186.

    Article  PubMed  CAS  Google Scholar 

  • Bergsma D, Desnick R, Bernlohr RW, and Krivit W. (eds) 1973. Enzyme therapy in genetic diseases. Baltimore: Williams & Wilkins for the National Foundation-March of Dimes, BD: OASIX (2).

    Google Scholar 

  • Bergstrom SK, Quinn JJ, Greenstein R, and Ascensao J. 1994. Long-term follow up of a patient transplanted for Hunter’s disease type IIB: a case report and literature review. Bone Marrow Trans. 14: 653–658.

    CAS  Google Scholar 

  • Beutler E. 1993. Gaucher disease as a paradigm of current issues regarding single gene mutations of humans. Proc. Natl. Acad. Sci. USA 90: 5384–5390.

    Article  PubMed  CAS  Google Scholar 

  • Birkenmeier EH, Davisson MT, Beamer WG, Ganschow RE, Vogler CA, Gwynn B, Lyford KA, Maltais LM, and Wawrzyniak CJ. 1989. Murine mucopolysaccharidosis type VII-characterization of a mouse with μ-glucuronidase deficiency. J. Clin. Invest. 83: 1258–66.

    Article  PubMed  CAS  Google Scholar 

  • Birkenmeier EH, Barker JE, Vogler CA, Kyle JW, Sly WS, Gwynn B, Levy B, and Pegors C. 1991. Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation. Blood. 78: 3081–3092.

    PubMed  CAS  Google Scholar 

  • Breider MA, Shull RM, and Constantopoulos G. 1989. Long-term effects of bone marrow transplantation in dogs with mucopolysaccharidosis I. Am J Pathol. 134: 677–692.

    PubMed  CAS  Google Scholar 

  • Brooks DA, McCourt PA, Gibson GJ, Ashton LJ, Shutter M, and Hopwood JJ. 1991. Analysis of N-acetylgalactosamine-4-sulfatase protein and kinetics in mucopolysaccharidosis type VI patients. Am. J. Hum. Genet. 48:710–719.

    PubMed  CAS  Google Scholar 

  • Chang PL, Shen N, and Westcott AJ. 1993. Delivery of recombinant gene products with microencapsulated cells in vivo. Hum. Gene Ther. 4: 433–440.

    Article  PubMed  CAS  Google Scholar 

  • Chang PL, Hortelano G, Tse M, and Awrey DE. 1994a. Growth of recombinant fibroblasts in alginate microcapsules. Biotech. Bioengin. 43: 925–933.

    Article  CAS  Google Scholar 

  • Chang PL, Lambert DT, and Pisa MA. 1994b. Behavioural abnormalities in a murine model of a human lysosomal storage disease. NeuroReport. 4: 507–510.

    Article  CAS  Google Scholar 

  • Chang PL. 1995. Non-autologous somatic gene therapy. In: Somatic gene therapy (Chang, PL, ed.) Boca Raton, Florida: CRC, Chap. 12.

    Google Scholar 

  • Clarke L, Russell CS, Pwnall S, Warrington CL, Borowski A, Dimmick JE, Toone J, and Jirik FR. 1997. Murine mucopolysaccharidosis type I: targeted disruption of the murine a-L-iduronidase gene. Hum. Mol. Gen. 6: 503–511.

    Article  PubMed  CAS  Google Scholar 

  • Cohen-Tannoudji M et al. 1995. Disruption of murine Hexa gene leads to enzymatic deficiency and to neuronal lysosomal storage, similar to that observed in Tay-Sachs disease. Mam. Genome. 6: 844–849.

    Article  CAS  Google Scholar 

  • Correll PH, Fink JK, Brady RO, Perry LK, and Karlsson S. 1989. Production of human glucocerebrosidase in mice after retoroviral gene transfer into multipotential hematopoietic progenitor cells. Proc. Nat. Acad. Sci. USA. 86: 8912–8916.

    Article  PubMed  CAS  Google Scholar 

  • Crawley AC, Brooks DA, Müller VJ, Petersen BA, Isaac EL, Bielicki J, King BM, Boulter CD, Moore AJ, Fazzalari NL, Anson DS, Byers S, and Hopwood JJ. 1996. Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome. J. Clin. Invest. 97: 1864–1873.

    Article  PubMed  CAS  Google Scholar 

  • Cunningham LA, Short MP, Vielkind U, Breakefield XO, and Bohn MC. 1991. Survival and differentiation within adult mouse striatum of grafted rat pheochromocytoma cells (PC 12) genetically modified to express recombinant β-NGF. Exp Neurobiol. 112: 174–182.

    Article  CAS  Google Scholar 

  • Desnick RJ, Dean KJ, Grabowski G, Bishop DF, and Sweeley CC. 1979. Enzyme therapy in Fabry disease-differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes. Proc. Natl. Acad. SCi. USA 76: 5326–5330.

    Article  PubMed  CAS  Google Scholar 

  • Evers M, Saftig P, Schmidt P, Hafner A, McLogin DB, Schmahl W, Hess B, von Figura K, and Peters, C. 1996. Proc. Nat. Acad. Sci. USA. 93: 8214–8219.

    Article  CAS  Google Scholar 

  • Fink JK, Correll PH, Perry LK, Brady RO, and Karlsson S. 1990. Correction of glucocerebroside deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with disease. Proc. Nat. Acad. Sci. USA. 87: 2334–2338.

    Article  PubMed  CAS  Google Scholar 

  • Food and Drug Administration 1991. FDA Med. Bull. 21:6–7.

    Google Scholar 

  • Gasper PW, Thrall MA, Wenger DA, Macy DW, Ham L, Dornsife RE, McBiles K, Quackenbush SL, Kesel ML, and Gillette EL. 1984. Correction of feline arylsulphatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation. Nature. 312: 467–469.

    Article  PubMed  CAS  Google Scholar 

  • Gieselmann V. 1995. Lysosomal storage disease. Biochim. Biophys. Acta Mol. Basis Dis. 1270: 103–136.

    Article  Google Scholar 

  • Ginns El, Choudary PV, Martin BM, Winnfield S, Stubblefield B, Mayor J, Merkle-Lehman D, Murray GJ, Bowers LA, and Barranger JA. 1984. Isolation of cDNA clones for human β-glucocerebrosidase using the Xgtl 1 expression system. Biochem. Biophys. Res. Comm. 123: 574–80.

    Article  PubMed  CAS  Google Scholar 

  • Grabowski GA. 1993. Gaucher disease: Enzymology, genetics, and treatment. Adv. Hum. Genet. 21: 377–441.

    PubMed  CAS  Google Scholar 

  • Hartley WJ, Canfield PJ, and Donnely TM. 1982. A suspected new canine storage disease. Acta Neuropathol. 56: 225–232.

    Article  PubMed  CAS  Google Scholar 

  • Haskins ME, Jezyk PF, Desnick RJ, McDonaugh SK, and Patterson DF. 1979. Alpha-L-iduronidase deficiency in a cat: a model of mucopolysaccharidosis I. Pediat. Res. 13: 1294–1297.

    Article  PubMed  CAS  Google Scholar 

  • Haskins ME, Aguirre GD, Jezyk PF, and Patterson DF. 1980. The pathology of the feline model of mucopolysaccharidosis VI. Am. J. Path. 101: 657–666.

    PubMed  CAS  Google Scholar 

  • Haskins ME, Desnick RJ, DiFerrante N, Jezyk PF, and Patterson DF. 1984. μ-glucuronidase deficiency in a dog: a model of mucopolysaccharidosis type VII. Pediatr. Res 18: 980–984.

    PubMed  CAS  Google Scholar 

  • Hatton JD, Lechtman AN, and U, HS. 1992. Formation of PC 12 tumors after implantation into rat brain: dependence of time course host stage. Cancer Res. 52: 1933–1937.

    PubMed  CAS  Google Scholar 

  • Hoffman D, Breakefield XO, Short MP, and Aebischer P. 1993. Transplantation of a polymer-encapsulated cell line genetically engineered to release NGF. Exp. Neurol. 122: 100–106.

    Article  PubMed  CAS  Google Scholar 

  • Hoogerbrugge PM, Brouwer OF, Bordigoni P, Ringden O, Kapaun P, Ortega JJ, O’Meara A, Cornu G, Souillet G, Frappaz. D, Blanche S, and Fischer A. 1995. Allogeneic bone marrow transplantation for lysosomal storage diseases. Lancet 345: 1398–1402.

    Article  PubMed  CAS  Google Scholar 

  • Hortelano G, Al-Hendy-A and Chang PL. 1996. Delivery of factor IX in mice using implantable microcapsules: towards gene therapy of hemophilia B. Blood 87: 5095–5103.

    PubMed  CAS  Google Scholar 

  • Jaeger CB. 1985. Immunocytochemical study of PC12 cells grafted into the brain of immature rats. Exp. Brain Res. 59: 615–624.

    Article  PubMed  CAS  Google Scholar 

  • Jaeger CB, Kapoor R, and Llinas R. 1988. Cytology and organization of rat cerebellar organ cultures. Neuroscience. 26: 509–538.

    Article  PubMed  CAS  Google Scholar 

  • Jezyk PF, Haskins ME, Patterson DF, Mellman WJ, and Greenstein M. 1977. Mucopolysaccharidosis in a cat with arylsulfatase B deficiency: a model of Maroteaux-Lamy syndrome. Science 198: 834–836.

    Article  PubMed  CAS  Google Scholar 

  • Krivit W, Pierpont ME, Ayaz K, Tsai M, Ramsay NK Kersey JH, Weisdorf S, Sibley R, Snover D, and McGovern MM. 1984. Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Biochemical and clinical status 24 months after transplantation. N. Engl. J. Med. 1984 311:1606–1611.

    Article  PubMed  CAS  Google Scholar 

  • Krivit W, and Whitley CB. 1987. Bone marrow transplantation for genetic diseases. New Engl. J. Med. 316: 1085–7.

    Article  PubMed  CAS  Google Scholar 

  • Krivit W, Whitley CB, Chang PN, Shapiro N, Belani KG, Snover D, Summers GC, and Blazar B. 1990. Lysosomal storage diseases treated by bone marrow transplantation: Review of 21 patients. In: Bone marrow transplantation in children (Johnson, FL and Pochedly, C, eds.) New York: Raven Press.

    Google Scholar 

  • Krivit W, Shapiro E, Hoogerbrugge PM, and Moser HW. 1992. State of the art review: bone marrow transplantation treatment for storage diseases. Bone Marrow Transplant. 10 Suppl. 1: 87–96.

    Google Scholar 

  • Kyle JW, Birkenmeier EH, Gwynn B, Vogler C, Hoppe PC, Hoffmann JW, and Sly WS. 1990. Correction of murine mucopolysaccharidosis VII by a human β-glucuronidase transgene. Proc. Natl. Acad. Sci. USA. 87: 3914–3918.

    Article  PubMed  CAS  Google Scholar 

  • Lacorazza HD, Flax JD, Snyder EY, and Jendoubi M. 1996. Expression of human beta-hexosaminidase alphasubunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells. Nat. Med. 2: 424–429.

    Article  PubMed  CAS  Google Scholar 

  • Ladisch S, Bayeuer E, Philipport M, and Feig S. 1986. Biochemical findings after bone marrow transplantation for metachromatic leukodystrophy: a preliminary report. Birth Defects. 22: 69–78.

    PubMed  CAS  Google Scholar 

  • Lau C, Soriano HE, Ledley FD, Finegold MJ, Wolfe JH, Birkenmeier EH, and Henning SJ. 1995. Retro viral gene transfer into the intestinal epithelium. Hum. Gene Ther. 6: 1145–1155.

    Article  PubMed  CAS  Google Scholar 

  • Li T, and Davidson BL. 1995. Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer. Proc. Natl. Acad. Sci. USA 92: 7700–7704.

    Article  PubMed  CAS  Google Scholar 

  • Liu H, Ofosu FA, and Chang PL. 1993. Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum. Gene Ther. 4: 291–301.

    Article  PubMed  CAS  Google Scholar 

  • Liu Y, Hoffmann A, Grinberg A, Westphal H, McDonald MP, Miller KM, Crawley JN, Sandhoff K, Suzuki K, and Proia RL. 1997. Mouse model of GM2 activator deficiency manifests cerebellar pathology and motor impairment. Proc. Natl. Acad. Sci. USA. 94: 8138–8143.

    Article  PubMed  CAS  Google Scholar 

  • Littlewood JD, Herrtage ME, and Palmer AC. 1983. Neuronal storage disease in English springer spaniels. Vet. Record. 112:86–87.

    Article  CAS  Google Scholar 

  • Marechal V, Naffakh N, Danos O, and Heard JM. 1993. Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells. Blood. 82: 1358–65.

    PubMed  CAS  Google Scholar 

  • McGovern MM, Mandell N, Haskins M, and Desnick RJ. 1985. Animal model studies of allelism: characterization of arylsulfatase B mutations in homoallelic and heteroallelic (genetic compound) homozygotes with feline mucopolysaccharidosis VI. Genetics. 110: 733–749.

    PubMed  CAS  Google Scholar 

  • Miyazano M, Lee VM, and Trojanowski JQ. 1995. Proliferation, cell death, and neural differentiation in transplanted human embryonal carcinoma (Ntera2) cells depend on the graft site in nude and SCID mice. Lab. Invest. 73: 273–283.

    Google Scholar 

  • Moullier P, Bohl D, Heard J-M, and Danos O. 1993. Correction of lysosomal storage in the liver and spleen of MPS VII affected mice by implantation of genetically modified skin fibroblasts. Nat. Gen. 4: 154–159.

    Article  CAS  Google Scholar 

  • Myerowitz R and Proia RL. 1984. cDNA clone for the a-chain of human B-hexosaminidase: deficiency of a-chain mRNA in Ashkenazi Tay-Sachs fibroblasts. Proc. Natl. Acad. Sci. 81: 5394–8.

    Article  PubMed  CAS  Google Scholar 

  • Naffakh N, Pinset C, Montarras D, Li Z, Paulin D, Danos O, and Heard J. 1996. Long term secretion of therapeutic proteins from genetically modified skeletal muscles. Hum. Gen. Ther. 7: 11–21.

    Article  CAS  Google Scholar 

  • Neufeld EF and Muenzer J. 1995. The mucopolysaccharidoses. In: The metabolic and molecular bases of inherited disease. (Scriver, CR, Beaudet, AL, Sly, WS, Valle, DV, eds. New York: McGraw-Hill Inc.

    Google Scholar 

  • Nimgaonkar, MT, Bahnson, AB, Mannion-Henderson, J, Barranger, JA, and Ball, ED. 1994. Hematopoietic stem cells as targets for gene therapy in Gaucher disease. Cold Spring Harbor Laboratory Meeting on Gene Therapy, p. 7, Sept, 1994.

    Google Scholar 

  • O’Dowd BF, Quan F, Willard HF, Lamhonwah AM, Korneluk RG, Lowden JA, Gravel RA, and Mahuran DJ. 1985. Isolation of cDNA clones coding for the beta subunit of human beta-hexosaminidase. Proc. Natl. Acad. Sci. USA. 82: 1184–8.

    Article  PubMed  Google Scholar 

  • Ohashi T, Watabe K, Uehara K, Sly WS, Vogler C, and Eto Y. 1997. Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc. Natl. Acad. Sci. USA. 94: 1287–1292.

    Article  PubMed  CAS  Google Scholar 

  • Oshima AJ, Kyle W, and Miller RD, Hoffmann JW, Powell PP, Grubb JH, Sly WS, Tropak M, Guise KS, and Gravel RA. 1987. Cloning, sequencing and expression of cDNA for human β-glucuronidase. Proc. Natl. Acad. Sci. 84: 685–9.

    Article  PubMed  CAS  Google Scholar 

  • Otterback B and Stoffel W. 1995. Acid sphingomyelinasedeficient mice mimic the neurovisceral form of human lysosomal storage disease (Niemann-Pick disease). Cell. 81: 1053–1061.

    Article  Google Scholar 

  • Parkman R. 1986. The application of bone marrow transplantation to the treatment of genetic diseases. Science. 232: 1373–7.

    Article  PubMed  CAS  Google Scholar 

  • Potter MA, Hymus SA, Stockley TL, and Chang PL. 1998. Suppression of immunological response against a transgene product delivered with microencapsulated cells. Hum. Gen. Ther. 9: 1275–1282.

    Article  CAS  Google Scholar 

  • Rappeport JM and Ginns El. 1984. Bone marrow transplantation in severe Gaucher’s disease. New Engl. J. Med. 311:84–8.

    Article  PubMed  CAS  Google Scholar 

  • Ross CJD et al. 1998. Allogeneic gene therapy of the central nervous system in the murine model of mucopolysaccharidosis type VII: Treatment and behavioral recovery. Manuscript to be submitted.

    Google Scholar 

  • Salvetti A, Moullier P, Cornet V, Brooks D, Heard JM, and Danos O. 1994. Preclinical studies for the in vivo delivery of human a-iduronidase in mucopolysaccharidosis type I. Cold Spring Harbor Meeting on Gene Therapy, September, 1994 p. 146.

    Google Scholar 

  • Salvetti A, Moullier P, Cornet V, Brooks D, Hopwood JJ, Danos O, and Heard JM. 1995. In vivo delivery of human alpha-L-iduronidase in mice implanted with neo-organs. Hum. Gene Ther. 6: 1153–1159.

    Article  PubMed  CAS  Google Scholar 

  • Sands MS, and Birkenmeier EH. 1993. A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII. Proc. Natl. Acad. Sci. U.S.A. 90: 6567–6571.

    Article  PubMed  CAS  Google Scholar 

  • Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, and Birkenmeier EH. 1994. Enzyme replacement therapy for murine mucopolysaccharidosis type VIL J. Clin. Invest. 93: 2324–31.

    Article  PubMed  CAS  Google Scholar 

  • Sands MS, Erway LC, Vogler C, Sly WS, and Birkenmeier EH. 1995. Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII. Blood. 86: 2033–2040.

    PubMed  CAS  Google Scholar 

  • Schinstine M, Fiore DM, Winn SR, and Emerich DF. 1995. Polymer-encapsulated schwannoma cells expressing human nerve growth factor promote the survival of cholinergic neurons after a fimbria-fornix transfection. Cell Transplant. 4: 93–102.

    Article  PubMed  CAS  Google Scholar 

  • Shull RM, Munger RJ, Spellacy E, Hall CW, Constantopoulos G, and Neufeld EF. 1982. Canine alpha-Liduronidase deficiency: a model of mucopolysaccharidosis I. Am. J. Path. 109: 244–248.

    PubMed  CAS  Google Scholar 

  • Shull RM, Xiaochen L, McEntree MF, Bright RM, Pepper KA, and Kohn DB. 1996. Myoblast gene therapy in canine MPS I: Abrogation by an immune response to a-L-iduronidase. Hum. Gene Ther. 7: 1596–1603.

    Article  Google Scholar 

  • Shull RM, Kakkis ED, McEntree MF, Kania SA, Jonas AJ, and Neufeld EF. 1994. Enzyme replacement in a canine model of Hurler syndrome. Proc. Nat. Acad. Sci. USA. 91: 12937–12941.

    Article  PubMed  CAS  Google Scholar 

  • Simonaro CM, Haskins ME, Kunieda T, Evans SM, Visser JW, and Schuchman EH. 1997. Bone marrow transplantation in newborn rats with mucopolysaccharidosis type VI: biochemical, pathological, and clinical findings. Transplantation. 63: 1386–1393.

    Article  PubMed  CAS  Google Scholar 

  • Sly WS, Quinton BA, McAlister WH, and Rimoin DL. 1973. ß-glucuronidase deficiency: report of clinical, radiologie, and biochemical features of a new mucopolysaccharidosis. J. Pediatr. 82: 249–257.

    Article  PubMed  CAS  Google Scholar 

  • Sly WS, and Vogler C. 1997. Gene therapy for lysosomal storage disease: a no-brainer? Transplants of fibroblasts secreting high levels of beta-glucuronidase decrease lesions in the brains of mice with Sly syndrome, a lysosomal storage disease. Nat. Med. 3: 719–720.

    Article  PubMed  CAS  Google Scholar 

  • Snyder EY, Taylor RM, and Wolfe JH. 1995. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature. 374: 367–70.

    Article  PubMed  CAS  Google Scholar 

  • Sorge J, West C, Westwood B, and Beutler E. 1985. Molecular cloning and nucleotide sequence of human glucocerebrosidase cDNA. Proc. Natl. Acad. Sci. 82: 7289–93.

    Article  PubMed  CAS  Google Scholar 

  • Spellacy E, Shull RM, Constantopoulos G, and Neufeld EF. 1983. A canine model of human alpha-L-iduronidase deficiency. Proc. Nat. Acad. Sci. 80: 6091–6095.

    Article  PubMed  CAS  Google Scholar 

  • Stein C, Gieselmann V, Kreysing J, Schmidt B, Pohlman R, Waheed A, and von Figura K. 1989. Cloning and expression of human arylsulfatase A. J. Biol. Chem. 264: 1252–9.

    PubMed  CAS  Google Scholar 

  • Taylor RM, Stewart GJ, and Farrow BRH. 1989. Improvement in the neurological signs and storage lesions of fucosidoses in dogs given marrow transplants at an early age. Transplant. Proc. 21: 3818–9.

    PubMed  CAS  Google Scholar 

  • Taylor RM, and Wolfe, JH. 1994. Cross-correction of ß-glucuronidase deficiency by retroviral vector-mediated gene transfer. Exp. Cell Res. 214: 606–613.

    Article  PubMed  CAS  Google Scholar 

  • Taylor RM, and Wolfe JH. 1997. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase. Nat. Med. 3: 771–774.

    Article  PubMed  CAS  Google Scholar 

  • Thomas GH. 1994. “Pseudodeficiencies” of lysosomal hydrolases. Am. J. Hum. Genet. 54: 934–940.

    PubMed  CAS  Google Scholar 

  • Thompson JN, Jones MZ, Dawson G, and Huffman PS. 1992. N-acetylglucosamine 6-sulphatase deficiency in a Nubian goat: a model of Sanfilippo syndrome type D (mucopolysaccharidosis HID). J. Inherit. Metab. Dis. 15: 760–768.

    Article  PubMed  CAS  Google Scholar 

  • Tresco PA, Winn SR, Tan S, Jaeger CB, Greene LA, and Aebischer P. 1992. Polymer encapsulated PC-12 cells: Long term survival and associated reduction in lesion induced rotational behavior. Cell Transplant. 1: 255–264.

    PubMed  CAS  Google Scholar 

  • Vandevelde M, Fankhauser R, Bichsel P, Wiesmann U, and Herschkowitz N. 1982. Hereditary neurovisceral mannosidosis associated with alpha-mannosidase deficiency in a family of Persian cats. Acta Neuropathol.(Berl.) 58: 64–68.

    Article  CAS  Google Scholar 

  • Vellodi A, Hobbs JR, O’Donnel NM, Coulter BS, and Hugh-Jones K. 1987. Treatment of Neiman-Pick disease type B by allogeneic bone marrow transplantation. Brit. Med. J. 295: 1375–1376.

    Article  CAS  Google Scholar 

  • Vellodi A, Young E, New M, Pot-Mees C, and Hugh-Jones K. 1992. Bone marrow transplantation for Sanfilippo disease type B. J. Inherit. Metab. Dis. 15: 911–918.

    Article  PubMed  CAS  Google Scholar 

  • Vogler C, Birkenmeier EH, Sly WS, Levy B, Pegors C, Kyle JW, and Beamer WG. 1990. A murine model of mucopolysaccharidosis VII-gross and microscopic findings in beta-glucuronidase-deficient mice. Am. J. Pathol. 136:207–17.

    PubMed  CAS  Google Scholar 

  • Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, and Sly WS. 1993. Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediatr. Res. 34: 837–840.

    Article  PubMed  CAS  Google Scholar 

  • Walkley SU, Thrall MA, Dobrenis K, Huang M, March PA, Siegel DA, and Wurzelmann S. 1994. BMT corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease. Proc. Nat. Acad. Sci. USA. 91: 2970–2974.

    Article  PubMed  CAS  Google Scholar 

  • Wilson JM. 1996. Animal models of human disease for gene therapy. J. Clin. Invest. 97: 1138–1141.

    Article  PubMed  CAS  Google Scholar 

  • Winn SR, Tresco PA, Zielinski B, Greene LA, Jaeger CB, and Aebischer P. (1991) Behavioral recovery following intrastriatal implantation of microencapsulated PC 12 cells. Exp. Neurobiol. 113: 322–329.

    Article  CAS  Google Scholar 

  • Wolfe, JH, Deshmane SL, and Fraser, NW. (1992a) Herpes virus vector gene transfer and expression of betaglucuronidase in the central nervous system of MPS VII mice. Nat. Genet. 1: 379–84.

    Article  PubMed  CAS  Google Scholar 

  • Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, Vogler CA, and Birkenmeier EH. (1992b) Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature. 360: 749–753.

    Article  PubMed  CAS  Google Scholar 

  • Yamanaka S, Johnson MD, Grinberg A, Westphal H, Crawley JN, Taniike M, Suzuki K, and Proia RL. (1994) Targeted disruption of the hexa gene results in mice with biochemical and pathological features of Tay-Sachs disease. Proc. Nat. Acad. Sci. USA. 91: 9975–9979.

    Article  PubMed  CAS  Google Scholar 

  • Yoshida M, Ikadai H, Maekawa A, Takahashi M, and Nagase S. (1993) Pathological characteristics of mucopolysaccharidosis VI in the rat. J. Comp. Pathol. 109: 141–153.

    Article  PubMed  CAS  Google Scholar 

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Authors
  1. Colin J. D. Ross
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  2. Patricia L. Chang
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Editors and Affiliations

  1. BioHybrid Technologies, Inc., Shrewsbury, MA, 05145, USA

    Willem M. Kühtreiber PhD  & William L. Chick MD  & 

  2. Tissue Engineering & Transplant Medicine, Advanced Cell Technology, Worcester, MA, 01605, USA

    Robert P. Lanza MD

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© 1999 Springer Science+Business Media New York

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Ross, C.J.D., Chang, P.L. (1999). Microencapsulation—A Novel Gene Therapy for Lysosomal Storage Diseases. In: Kühtreiber, W.M., Lanza, R.P., Chick, W.L. (eds) Cell Encapsulation Technology and Therapeutics. Birkhäuser, Boston, MA. https://doi.org/10.1007/978-1-4612-1586-8_26

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  • DOI: https://doi.org/10.1007/978-1-4612-1586-8_26

  • Publisher Name: Birkhäuser, Boston, MA

  • Print ISBN: 978-1-4612-7205-2

  • Online ISBN: 978-1-4612-1586-8

  • eBook Packages: Springer Book Archive

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