Targeting disorders

ACE inhibitors are widely prescribed for patients with atherosclerosis, hypertension, or diabetes, and after myocardial infarction, because of proven beneficial effects in each of these groups. In light of the results in the setting of stent restenosis, whether a patient with hypertension or some other target disorder who carries the DD genotype derives less benefit from ACE inhibitor therapy warrants consideration. 

Phase II Assess drug s effectiveness in treating a specific disease/disorder Limited number (200-300) patients with target disorder 2 yrs... 

Onodera M, Sakiyama Y (2000). Adenosine deaminase deficiency as the first target disorder in gene therapy. Expert Opin. Investig. Drugs. 9 543-549. 

In order to examine whether this sequence gave a fold similar to the template, the corresponding peptide was synthesized and its structure experimentally determined by NMR methods. The result is shown in Figure 17.15 and compared to the design target whose main chain conformation is identical to that of the Zif 268 template. The folds are remarkably similar even though there are some differences in the loop region between the two p strands. The core of the molecule, which comprises seven hydrophobic side chains, is well-ordered whereas the termini are disordered. The root mean square deviation of the main chain atoms are 2.0 A for residues 3 to 26 and 1.0 A for residues 8 to 26. 

Caneer is a disorder of the body s eontrol of die growth of eells. The disease may be genetie or influeneed by life style or exposure to eertain ehemieals, termed eareinogens. For a list of examples of human ehemieal eareinogens, and the relevant target organs, refer to Table 5.10. 

Insulin is a peptide hormone, secreted by the pancreas, that regulates glucose metabolism in the body. Insufficient production of insulin or failure of insulin to stimulate target sites in liver, muscle, and adipose tissue leads to the serious metabolic disorder known as diabetes mellitus. Diabetes afflicts millions of people worldwide. Diabetic individuals typically exhibit high levels of glucose in the blood, but insulin injection therapy allows diabetic individuals to maintain normal levels of blood glucose. 

Little attempt has been made to develop drugs targeted specifically to white adipose tissue and the production of adipokines. It is likely, however, that there will be an increasing emphasis on this approach to the pharmacological treatment of obesity-related diseases, given the current views on the centrality of the adipokines to these disorders. It is, of course, the diseases that obesity leads to, rather than obesity itself, that constitute the main medical challenge. 

Antisense Oligonucleotides. Table 1 Malignant disorders as potential targets for ribozyme gene therapy... 

However, despite its enormous importance to human physiology, no pharmacological compounds targeting the components of the circadian clock system have been identified to date. There are, nevertheless, two therapeutic approaches that are currently used for treatment of circadian-related disorders - full-spectrum and bright light therapy and melatonin therapy. Melatonin is a hormone that is produced by the pineal gland in... 

Holmes A, Picciotto MR (2006) Galanin a novel therapeutic target for depression, anxiety disorders and drag addiction CNS Neurol Disord Drag Targets 5 225-232... 

MC4R for feeding behaviour, targeting one or both of these recqDtors in addition to understanding the complex cross-talk within the CNS may lead to the development of melanocortin based therapeutics for the treatment of eating disorders [5]. 

Smith CA, Baker EN (2002) Aminoglycoside antibiotic resistance by enzymatic deactivation. Curr Drug Targets Infect Disord 2 143-160... 

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