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Titolo:
Enzyme replacement therapy in feline mucopolysaccharidosis I
Autore:
Kakkis, ED; Schuchman, E; He, X; Wan, Q; Kania, S; Wiemelt, S; Hasson, CW; OMalley, T; Weil, MA; Aguirre, GA; Brown, DE; Haskins, ME;
Indirizzi:
Harbor UCLA Med Ctr, Dept Pediat, Div Med Genet, Torrance, CA 90502 USA Harbor UCLA Med Ctr Torrance CA USA 90502 d Genet, Torrance, CA 90502 USA CUNY Mt Sinai Sch Med, Dept Genet, New York, NY 10029 USA CUNY Mt Sinai Sch Med New York NY USA 10029 Genet, New York, NY 10029 USA Cornell Univ, Coll Vet Med, Ithaca, NY USA Cornell Univ Ithaca NY USACornell Univ, Coll Vet Med, Ithaca, NY USA Colorado State Univ, Dept Pathol, Ft Collins, CO 80523 USA Colorado State Univ Ft Collins CO USA 80523 hol, Ft Collins, CO 80523 USA Univ Penn, Sch Vet Med, Dept Pathobiol, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 Pathobiol, Philadelphia, PA 19104 USA
Titolo Testata:
MOLECULAR GENETICS AND METABOLISM
fascicolo: 3, volume: 72, anno: 2001,
pagine: 199 - 208
SICI:
1096-7192(200103)72:3<199:ERTIFM>2.0.ZU;2-K
Fonte:
ISI
Lingua:
ENG
Soggetto:
ALPHA-L-IDURONIDASE; BETA-GLUCURONIDASE; LYSOSOMAL-ENZYME; HUMAN FIBROBLASTS; GAUCHER DISEASE; CANINE MODEL; MPS-I; RECOGNITION; CELLS; VII;
Keywords:
lysosomal storage disorder; glycosaminoglycan; immune response; alpha-L-iduronidase; mucopolysaccharidosis; animal model; cat;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
31
Recensione:
Indirizzi per estratti:
Indirizzo: Kakkis, ED BioMarin Pharmeceut Inc, 371 Bel Marin Keys Blvd,Suite 210, Novato, CA 94949 USA BioMarin Pharmeceut Inc 371 Bel Marin Keys Blvd,Suite 210Novato CA USA 94949
Citazione:
E.D. Kakkis et al., "Enzyme replacement therapy in feline mucopolysaccharidosis I", MOL GEN MET, 72(3), 2001, pp. 199-208

Abstract

Enzyme replacement therapy (ERT) has long been considered an approach to treating lysosomal storage disorders caused by deficiency of lysosomal enzymes. ERT is currently used to treat Graucher disease and is being developed for several lysosomal storage disorders now that recombinant sources of theenzymes have become available. We have continued development of ERT for mucopolysaccharidosis I (MPS I) using the feline model, Recombinant alpha -L-iduronidase was administered intravenously at low dose (similar to0.1 mg/kgor 25,000 units/kg) to four cats and high dose (0.5 mg/kg or 125,000 units/kg) to two cats on a weekly basis for 3- or g-month terms, Clinical examinations showed distinct clearing of corneal clouding in one cat although clinical effects in the others were not evident. Biochemical studies of the cats showed that the enzyme was distributed to a variety of tissues although the liver and spleen contained the highest enzyme activities. Glycosaminoglycan storage was decreased in liver and spleen, and the histologic appearance improved in liver, spleen, and renal cortex. Enzyme was not consistentlydetected in cerebral cortex, brainstem, or cerebellum and the histologic appearance and ganglioside pro files did not improve. A variety of other tissues showed low variable uptake of enzyme and no distinct improvement, IgG;antibodies to alpha -L-iduronidase were observed in five cats with higher titers noted when higher doses were administered. Mild complement activation occurred in three cats, Enzyme replacement therapy was effective in reversing storage in some tissues at the biochemical and histologic level in MPSI cats but an improved tissue distribution and prevention of a significantimmune response could make the therapy more effective. (C) 2001 Academic Press.

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Documento generato il 09/04/20 alle ore 00:22:32