PRECURSOR N-ACETYLGALACTOSAMINE-4-SULFATASE, METHODS OF TREATMENT USING SAID ENZYME AND METHODS FOR PRODUCING AND PURIFYING SAID ENZYME

The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N -acetylgalactosamirie-4-sulfat...

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Hauptverfasser: LIN CHEN, PAUL, A. FITZPATRICK, STUART SWIEDLER, WAI-PAN CHAN, GARY N. ZECHERLE, DAN J. WENDT, JOHN M. HENSTRAND, MINMIN QIN, CHRISTOPHER M. STARR
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Sprache:eng ; por
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Zusammenfassung:The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N -acetylgalactosamirie-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N -acetylgalactosamine-4-sulfatase including MPS Vl and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.