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

• Main Author: STUART SWEIDLER
• Format: Patent
• Language: English; Spanish
• Published: 22.07.2005
• Edition: 7
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

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-acetylgalactosamine-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 VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form. La presente invencion proporciona una N-acetilgalactosamina-4-sulfatasa precursora recombinante humana altamente purificada y mutantes, fragmentos y analogos biologicamente activos de la misma asi como formulaciones farmaceuticas que comprenden N-acetilgalactosamina-4-sulfatasa precursora recombinante humana. La invencion tambien proporciona metodos para tratar enfermedades ocasionadas totalmente o en parte por deficiencias en N-acetilgalactosamina-4-sulfatasa humana incluyendo MPS VI y metodos para producir y purificar la enzima precursora recombinante a una forma altamente purificada.