Protalix's development pipeline also includes the following product candidates: PRX-102, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; PRX-105, a pegylated recombinant human acetylcholinesterase in development for several therapeutic and prophylactic indications, a biodefense program and an organophosphate-based pesticide treatment program; an orally-delivered
glucocerebrosidase enzyme that is naturally encased in carrot cells, also for the treatment of Gaucher disease; pr-antiTNF, a similar plant cell version of etanercept (Enbrel) for the treatment of certain immune diseases such as rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis; and others.
* Baseline enzyme activity of
glucocerebrosidase in leukocytes
Glucocerebrosidase enzyme level was measured as 1.6 nmol/s/mpgr (5-13.5 nmol/s/mpgr).
(11) This mutation results in a deficiency in
glucocerebrosidase, a lysosomal enzyme.
Gaucher disease Type I is a disorder caused by a lack of
glucocerebrosidase, an enzyme that helps clear glucocerebroside from cellular structures called lysosomes.
Special stains (periodic acid-Schiff with diastase, Congo red, and colloidal iron) and presence of polarizable material can help rule out foreign material, such as silicone and polyvinylpyrrolidone (PVP).[1] Gaucher disease has long been confused with CSH, although Gaucher cells have paler, so-called wrinkled-paper cytoplasm.[3,4] Clinical features of splenomegaly and an abnormal
glucocerebrosidase level are diagnostic.
He spent the next nine years purifying the enzyme
glucocerebrosidase from human placentas.
(Tay-Sachs disease is probably the best-known of these.) Gaucher disease is caused by a deficiency of the enzyme
glucocerebrosidase, which is responsible for breaking down and digesting glucocerebroside, a by-product of the normal recycling of red blood cells.
Gaucher disease
Glucocerebrosidase Neimann-Pick Sphingomyelinase Tay-Sachs [Beta] hexosaminidase Sandhoff [Beta] hexosaminidase Fabry [Alpha]-galactosidase Wolman Acid esterase Generalized gangliosidosis [Beta]-galactosidase
The recent arrival on the market of a modified
glucocerebrosidase (Ceredase) for Gaucher's Disease and last year's approval of PEG-adenosine deaminase for severe combined immunodeficiency disease illustrate the growing capabilities of the pharmaceutical industry to provide endogenous compounds that patients are lacking.