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Replacing Missing Proteins

Several recombinant drugs treat inherited conditions by replacing missing or malfunctioning proteins. Several of these are enzymes, proteins that drive biochemical reactions. The body has many different enzyme proteins, each of which performs a specific job in the construction or breakdown of chemicals. Some inherited conditions are caused by a destructive buildup of substances, a buildup that occurs because of an error in the gene for a particular enzyme that breaks down that chemical. [Pg.62]

One example is an enzyme replacement drug used to treat a form of Gaucher disease, which is caused by an inherited error in the gene coding for the enzyme that breaks down the fatty substance cerebroside. In Gaucher disease, certain immune system cells [Pg.62]

The placenta-derived enzyme was treated to make any associated risk of viral infections low, but a small risk of infection from human tissue remained, so scientists developed a recombinant replacement enzyme, with the right sugar embellishment, produced from a laboratory source. Unfortimately, about 15% of patients treated with the replacement enzyme develop antibodies to the protein that can reduce the usefulness of the injections and pose a risk of mild to severe allergic reactions. At the moment, however. [Pg.63]

Like many recombinant treatments for chronic diseases, the recombinant replacement enzyme for Gaucher disease is very expensive A year s treatment can cost over 150,000, though most private insurance companies will reimburse patients for its use. [Pg.64]


Early development of nucleic acid therapy was aimed at genetic deficiency diseases which were amenable to replacement therapy, or correction by administration of the protein products of the defective genes, as described in Chapter 3. The introduction of the normal counterpart of a defective gene into cells of the patient, resulting in the long-term production of the missing protein, appeared as a desirable alternative to the repeated parenteral administration of purified protein (Verma, 1990 Caskey, 1992). However, the current span of nucleic acid-based therapies far exceeds this limited application conceived initially (Riordan and Martin, 1991 Crooke, 1992a). [Pg.198]

The complete identification of the amino acids which are essential in the diet is due to W.C. Rose (1938). His first attempts to replace casein with its constituents were unsuccessful because an essential amino acid component in the protein hydrolysate had been missed. After threonine had been isolated by him from casein and fibrin, and shown to be essential, Rose identified val, met, his, lys, phe, leu, ile, thr, and arg as... [Pg.24]

In contrast to methods involving single substitutions, combinatorial strategies are ideally suited for evaluating the complex network of interactions in proteins that ultimately determine form and function [73]. As shown in the mechanistic and structural investigations of chorismate mutases, degenerate protein ensembles can illuminate subtle trends that might be missed in analyses of individual amino acid replace-... [Pg.57]


See other pages where Replacing Missing Proteins is mentioned: [Pg.62]    [Pg.673]    [Pg.62]    [Pg.673]    [Pg.84]    [Pg.35]    [Pg.239]    [Pg.412]    [Pg.406]    [Pg.175]    [Pg.98]    [Pg.38]    [Pg.32]    [Pg.426]    [Pg.167]    [Pg.70]    [Pg.74]    [Pg.19]    [Pg.414]    [Pg.98]    [Pg.52]    [Pg.85]    [Pg.175]    [Pg.409]    [Pg.1172]    [Pg.1480]    [Pg.728]    [Pg.192]    [Pg.233]    [Pg.501]    [Pg.366]    [Pg.526]    [Pg.125]    [Pg.434]    [Pg.334]    [Pg.565]    [Pg.181]    [Pg.728]    [Pg.799]    [Pg.179]    [Pg.263]    [Pg.209]    [Pg.219]    [Pg.624]    [Pg.1218]    [Pg.115]    [Pg.33]    [Pg.391]   


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