Übersetzung für "Speicherkrankheit" in Englisch

WOMAN: Both indicate lysosomal storage illness.

We have initiated a clinical trial to treat a lysosomal storage disease.

GM1 gangliosidosis is a lysosomal storage disease that leads to neurological disorders.

Mucopolysaccharidosis type MPS I-S represents a rare, autosomal-recessive inherited, metabolic disease with storage characteristics.

If you suffer from the MPS VI storage disorder, the reduced or absent N-acetylgalactosamine 4-sulfatase activity results in the accumulation of substrates in most cell types and tissue.

Globoid cell leukodystrophy (GLD, Krabbe disease) is a severe lysosomal inherited storage disease caused by insufficient function of galactocerebrosidase (GALC).

To understand, what a lysosomal storage disease is, we must have a look at an animal cell and will try to figure out, what 'lysosomal' and 'gangliosidose' and 'storage' mean.

Gangliosidose is a so called lysosomal storage disease, a so called enzymopathy (= a defect in the production of a necessary enzyme).

A biotherapeutic complex for use in a method of treating a subject suffering from a lysosomal storage disease (LSD), wherein the method comprises administering to the subject the biotherapeutic complex and wherein the biotherapeutic complex enters a lysosome of a cell of the subject and provides an enzyme activity ("replacement enzyme") that replaces the enzymatic activity that is associated with the LSD ("endogenous enzyme"), wherein the biotherapeutic complex comprises:

Background information: The forerunner of the Alpha-Man project was the "Euroman" project, which ran from 2001 to 2004 and received 1.6 million euros in support from the European Union. The project was a systematic and multi-discipline approach to research and therapy of the hereditary lysosomal storage disorder Alpha-mannosidosis.

Backround: Mucopolysaccharidosis (MPS) type III-A represents a rare, autosomal-recessive inherited, metabolic disease with lysosomal storage characteristics.

The Lagotto storage disease (LSD) is a storage disease with neurodegenerative symptoms, which leads to cerebellar damage in affected animals.

That means, if the amount of Gangliosids stored in the neurons is too high, i.e. the Gangsliosids cannot be destructed, than it is called a "storage disease".

Mucopolysaccharidosis Type VI is a lysosomal storage disease caused by a deficiency of N-acetylgalactosamine-4-sulfatase (4S).

Alpha-mannosidosis is a lysosomal storage disease that is caused by the deficiency of lysosomal alpha-mannosidase.

The cargo is alpha-Galactosidase (a-Gal), a therapeutic enzyme used for treatment of Fabry disease, a genetic lysosomal storage disorder22.

It is used for enzyme replacement therapy in Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome), a storage disorder where the level of N- acetylgalactosamine 4-sulfatase enzyme activity is absent or lower than normal.

In the year 2000 a Shiba was diagnosed for the first time in Japan with a rare hereditary disease that was known in different breeds but until then had not occurred in Shibas. This concerns a so-called storage disorder (metabolic disease) by the name of GM1 gangliosidosis which is not curable and is fatal within one and a half years.

When the radioactive label is incorporated on the NC cargo, the described parameters reflect transport of the cargo, which in our example was a-Gal enzyme, used for treatment of a genetic lysosomal storage disorder known as Fabry disease (Table 1). In addition to calculating NC transport by tracing said cargo, transepithelial delivery of this therapeutic enzyme can be estimated, e.g.by expressing it as molecules or mass of a-Gal transported per cell.

Besides these hereditary diseases common to humans and dogs, some less frequent diseases exist which have occurred only in single breeds, for example a certain kind of inherited renal cancer developed only in German Shepherd Dogs. Also the fatal storage disease gangliosidosis until now has been reported to have affected only the Shiba, and 7 further breeds.