alpha-l-iduronidase

(redirected from a-l-iduronidase)

α-l-i·dur·on·id·ase

(ī'dūr-on'i-dās),
An enzyme that hydrolyzes terminal desulfated α-l-iduronic acid residues of dermatan sulfate and of heparan sulfate; a deficiency of this enzyme is associated with Hurler syndrome and Scheie syndrome.

alpha-L-iduronidase

deficiency of the enzyme considered to be counterpart of mucopolysaccharidosis in cats and dogs; a neuronal storage disease.
References in periodicals archive ?
REGENXBIO was granted Orphan Drug Designation by the FDA for its investigational gene therapy RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I), a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene.
Cord blood has been proposed as an alternative stem cell source for children with HS since it has been suggested that cord blood may increase their levels of lysosomal a-L-iduronidase, which consequently may allow them to live longer with fewer complications," said lead study author jaap Jan Boelens, M.
Ninety-eight percent of the cord blood recipients whose transplanted cells were successfully engrafted had normal enzyme levels, supporting a link between high levels of lysosomal a-L-iduronidase in cord blood and suggested improvements in long-term outcomes among HS patients who receive UCB transplants.
Note that IDS-P and the substrate for a-L-iduronidase (for assay of MPS-I) (4) are structurally similar, but because they differ in the number of methylene groups in the hydrophobic linker, it should be straightforward to analyze for MPS-I and -II in the same infusion into the mass spectrometer.
a-L-Iduronidase, R-D-glucuronidase, and 2-sulfo-L-iduronate 2-sulfatase: preparation and characterization of radioactive substrates from heparin.
Recombinant human a-L-iduronidase has been studied as an enzyme replacement therapy with follow-up data available for the first 2 years.
and Genzyme General (NASDAQ:GENZ) intend to form a joint venture to develop and commercialize BioMarin's lead product, a-L-iduronidase, a recombinant enzyme designed to treat the genetic disorder known as mucopolysaccharidosis I (MPS I).
BioMarin initiated a pivotal clinical trial of a-L-iduronidase in January, following the collection of positive pre-clinical data.
Characterized by a halt in a patient's physical and mental development, MPS I is caused by lack of an active enzyme, a-L-iduronidase, which results in a build-up of certain carbohydrate materials in all parts of the body.
Neufeld and Kakkis developed a method for producing the recombinant form of a-L-iduronidase.
Our data suggest that many of the clinical problems associated with MPS I patients -- such as enlarged liver and spleen, airway obstruction and joint stiffness -- may be effectively addressed with enzyme replacement therapy using recombinant a-L-iduronidase.
Characterized by a halt in the patient's physical and mental development, the disease is caused by lack of an active enzyme, a-L-iduronidase, which results in a build-up of certain carbohydrate materials in all parts of the body.