Synthetic alpha -L-iduronidase and genetic sequences encoding same

摘要

The present invention relates generally to alpha -L-iduronidase and to genetic sequences encoding same. More particularly, the present invention provides an isolated nucleic acid molecule comprising a sequence of nucleotides which encodes or are complementary to a sequence which encodes a mammalian alpha -L-iduronidase or fragment or derivative thereof and to the recombinant enzyme encoded thereby. These molecules are useful in the investigation, diagnosis and treatment of subjects suspected of or suffering from alpha -L-iduronidase deficiency.