| abstract |
"N-ACETILGALACTOSAMINE-4-SULFATASE PRECURSOR, TREATMENT USING ENZYME METHODS AND METHODS FOR ENZYME PRODUCTION AND PURIFICATION". The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and its biologically active mutants, fragments or analogs, as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused in whole or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase, including MPS VI, and methods for producing and purifying the recombinant precursor enzyme to produce a highly purified form. . |