Title: Alglucerase
CAS Registry Number: 143003-46-7
CAS Name: Glucosylceramidase (human placental isoenzyme protein moiety reduced)
Additional Names: mannose-terminated human placental glucocerebrosidase; macrophage-targeted b-glucocerebrosidase
Trademarks: Ceredase (Genzyme)
Literature References: Modified form of b-glucocerebrosidase, an enzyme involved in glycolipid metabolism, which is deficient in patients with Gaucher's disease. Designed to target the tissue macrophages in which the lipid accumulates. Monomeric glycoprotein of 497 amino acids. Prepd by sequential deglycosolation of the native placental-derived enzyme to expose terminal mannose residues that can be recognized by macrophage cell surface receptors. Prepn: F. S Furbish et al., Biochim. Biophys. Acta 673, 425 (1981); G. J. Murray, Methods Enzymol. 149, 25 (1987). Clinical evaluation in Gaucher's disease: N. W. Barton et al., N. Engl. J. Med. 324, 1464 (1991); M. L. Figueroa et al., ibid. 327, 1632 (1992). Review of pharmacology and therapeutic efficacy: R. Whittington, K. L. Goa, Drugs 44, 72-93 (1992). Effect of long-term treatment: N. J. Weinreb et al., Am. J. Med. 113, 112-119 (2002).
Derivative Type: Imiglucerase
Additional Names: RGCR
Trademarks: Cerezyme (Genzyme)
Literature References: Mannose-terminated human glucocerebrosidase produced by recombinant DNA technology. Prepn: J. Rasmussen et al., WO 9007573; eidem, US 5236838 (1990, 1993 both to Genzyme).
Therap-Cat: Enzyme replacement therapy for Gaucher's disease.
Keywords: Enzyme Replacement Therapy. |