Therapies, enzyme

Thrombolytic Enzymes. Although atherosclerosis and the accompanying vascular wall defects are ultimately responsible for such diseases as acute pulmonary embolism, arterial occlusion, and myocardial infarction, the lack of blood flow caused by a fibrin clot directly results in tissue injury and in the clinical symptoms of these devastating diseases (54). Thrombolytic enzyme therapy removes the fibrin clot by dissolution, and has shown promise in the treatment of a number of thrombo-occlusive diseases (60). 

Frankenberg L. 1980. Enzyme therapy in cyanide poisoning Effect of rhodanese and sulfur compounds. Arch Toxicol 45(4) 315-323. 

Although most of the enzyme-based drugs are inhibitors of enzymes, a number of enzyme preparations have also been developed as drugs for the treatment of a number of diseases. The development of enzymes as therapeutics has been made easier due to the advances in biotechnology. Most successful example of enzyme therapy includes various preparations of plasminogen activators (thrombolytic or fibrinolytic agents) such as a bacterial protein streptokinase and two plasminogen activators... 

The availability of endogenous enzymes and their variants has allowed the use of enzymes as replacement therapy and as therapeutic agents. With continued refinement in recombinant protein technology, the cost of human recombinant proteins may become more affordable. As more novel human recombinant enzymes are developed, a wide variety of medical disorders will be amenable to enzyme therapies. Additional molecular engineering such as pegylation or creation and modification of... 

Grabowski, G.A., N.W. Barton, G. Pastores, J.M. Dambrosia, T.K. Banerjee, M.A. McKee, C. Parker, R. Schiffmann, S.C. HiU, and R.O. Brady, Enzyme therapy in type 1 Gaucher disease comparative efficacy of mannose-terminated glucocerebrosidase... 

Fabry disease is an X-linked disorder and accurate carrier detection is important for genetic counseling and because enzyme therapy is available for clinically affected hemizygote and heterozygote patients. Because of random X-inactivation, not all heterozygotes can be reliably identified by enzyme assay, and mutation analysis is strongly recommended. 

Enzyme Therapy in Genetic Diseases," Birth Defects Original Article Series, Vol. IX (Bergsma, D., ed.) The Williams and Wilkins Co., Baltimore (1973). 

E Nassif, M Younoszai, M. Weinberger, and C. Nassif. Comparative effects of antacids, enteric coating, and bile salts on (he efficacy of oral pancreatic enzyme therapy in cynic fibrosis. /. Pedialr, 9ft320 (1981). 

A. Nagi, J. R. DeLoach, K. Andrews, etal. Determination of parameters for enzyme therapy using l-asparaglnase entrapped in canine erythrocytes. BiotechaoL Appl Biochem. 70 365 (1988). 

Cornell HJ, Macrae FA, Melny J, Pizzey CJ, Cook F, Mason S, et al. Enzyme therapy for management of coeliac disease. Scand J Gastroenterol 2005 40 1304-1312. 

Pastores GM, Sibille AR, Grabowski GA. Enzyme therapy in Gaucher disease type 1 dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months. Blood 1993 82(2) 408-16. 

Another, more recent, example is enzyme therapy for Gaucher s disease, an inborn error of metabolism, treated with Ceredase. The therapy, which requires more than a ton of placenta annually to extract and make the drug, can cost as much as 500,000 per year per person, depending on the dosage needed. A 1996 National Institutes of Health technology assessment panel addressed issues in diagnosis and treatment of the disease and concluded that despite the success of enzyme therapy, treatment is limited by the cost. 

Carroccio A, Guarino A, Zuin G, Verghi F, Canani RB, Fonatana M, Bruzzese E, Montalto G, Notarbartolo A. Efficacy of oral pancreatic enzyme therapy for the treatment of fat malabsorption in HIV-infected patients. Aliment Pharmacol Ther 2001 15 25(2). 

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