LYSOSOMAL STORAGE DISEASE ENZYME

摘要

PROBLEM TO BE SOLVED: To provide lysosomal storage disease enzyme.SOLUTION: The invention provides: a composition comprising a recombinant human lysosomal acid lipase having a particular glycosylation pattern for internalization into the target cell; a vector containing a nucleic acid encoding human lysosome acid lipase; a host cell transformed with the vector; a pharmaceutical composition comprising the recombinant human lysosomal acid lipase; and a method for treating conditions associated with the lysosomal acid lipase deficiency. For example, provided is a composition comprising an isolated human recombinant lysosomal acid lipase (LAL) comprising one or more N-glycan structures.