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
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). 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.