Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

• Main Authors: PAUL A. FITZPATRICK, WIA-PAN CHAN, LIN CHEN, BECKY TANAMACHI, EMIL D. KAKKIS, DAN J. WENDT, GARY N. ZECHERLE, JOHN M. HENDSTRAND, MINMIN QIN, CHRISTOPHER M. STARR
• Format: Patent
• Language: English
• Published: 03.06.2004
• 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; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

The present invention provides a recombinant human ±-L-iduronidase and biologically active fragments and mutants thereof, large scale methods to produce and purify commercial grade recombinant human, ±-L-iduronidase enzyme as well as methods to treat certain genetic disorders including ±-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).