C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 9/24 (2006.01) A61K 38/47 (2006.01) A61K 47/34 (2006.01) A61P 3/00 (2006.01) C07K 1/36 (2006.01) C12P 21/02 (2006.01)
Patent
CA 2391098
The present invention provides a recombinant human .alpha.-L-iduronidase and biologically active fragments and mu-tants thereof, large scale methods to produce and purify commercial grade recombinant human .alpha.-L-iduronidase enzyme as well as methods to treat certain genetic disorders including .alpha.-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).
L'invention concerne une alpha -L-iduronidase recombinante humaine et des fragments et des mutants actifs sur le plan biologique de celle-ci, ainsi que des procédés à grande échelle de préparation et de purification de qualité commerciale d'enzymes d' alpha -L-iduronidase recombinante humaine et des méthodes de traitement de certaines maladies génétiques, notamment une déficience de l' alpha -L-iduronidase et la mucopolysaccharidose I (MPS 1).
Chan Wai-Pan
Chen Lin
Fitzpatrick Paul A.
Henstrand John M.
Kakkis Emil D.
Biomarin Pharmaceutical Inc.
Gowling Lafleur Henderson Llp
Harbor-Ucla Research And Education Institute
Los Angeles Biomedical Research Institute At Harbor-Ucla Medical
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