U.S. Patent 6,149,909, entitled "Synthetic Alpha-L-Iduronidase and Genetic Sequences Encoding Same," based on work performed by John Hopwood and colleagues, has recently been issued. The patent claims are related to the purified alpha-L-iduronidase made from cells transfected with DNA encoding the protein. The protein may be useful in the investigation, diagnosis, and treatment of Hurler syndrome, also know as mucopolysaccharidosis I (MPS I). The patent was issued to Women's and Children's Hospital (North Adelaide, Australia) and licensed to Transkaryotic Therapies (9195 Albany St., Cambridge, MA 02139; Tel: 617/349-0200, Fax: 617/491-7903) through an exclusive worldwide license agreement.
"We are committed to developing treatments for rare genetic disorders, and Hopwood's work offers real hope to patients with Hurler, Hurler-Scheie, and Scheie syndromes," states Richard F. Selden, president and CEO of Transkaryotic Therapies.
MPS I is an inherited rare genetic disorder caused by deficient activity of the protein alpha-L-iduronidase. The enzyme is present in lysosomes, where it helps break down mucopolysaccharides. In patients affected by MPS I, these mucopolysaccharides accumulate in organs and tissues of the body, particularly in the central nervous system, liver, spleen, heart, and skeleton, ultimately lending to cell death and progressive tissue and organ damage. About 1 in 25,000 births will result in some form of MPS in the United States.
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