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AssigneeWomen's and Children's Hospital
InventorsHamish Steele Scott, Donald Stewart Anson, Annette Marie Orsborn, Paul Victor Nelson, Peter Roy Clements, Charles Phillip Morris, John Joseph Hopwood
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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.
A purified .alpha.-L-iduronidase (IDUA) wherein the IDUA is secreted and purified from mammalian cells in culture transfected with a DNA sequence encoding human IDUA.
A pharmaceutical composition comprising purified .alpha.-L-iduronidase (IDUA) and one or more pharmaceutically acceptable carriers or diluents wherein said IDUA is secreted and purified from mammalian cells in culture transfected with a DNA sequence encoding human IDUA.
Dependencies are inferred from the claim text.