RECOMBINANT agr;-L-IDURONIDASE, METHODS FOR PRODUCING AND PURIFYING THE SAME AND METHODS FOR TREATING DISEASES CAUSED BY DEFICIENCIES THEREOF.

摘要

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