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Targeted drug delivery (Site specific drug delivery)

机译:有针对性的药物输送(特定地点的药物输送)

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The conventional drugs used so far are small organic molecules meant to degrade the diseased protein, formed as a result of the interference of pathogens at cellular level. A high concentration of these drugs in the cells is necessary for desired therapeutic effect. This aggravates the problem of toxicity. Ever since the unraveling of human genome, attempts are being made to target the drugs selectively to the affected sites in order to increase their efficiency and simultaneously decrease their side effects. 'Proteomics' and 'genomics' have emerged as new disciplines. The 'target identification' for any ligand (drug) can now be achieved by identification of 'right gene', 'right pathway' and 'right target,' and the 'right drug' for 'right patient' can be developed. Genomics information decides the target validation, which is, in fact the association between particular target and disease process. Target validation can be achieved either at DNA level using gene knockout strategies and triplex formation, at RNA level using RNA interference (RNAi), at m-RNA level using antisense concept, at ribozyme level or at any other intermediate growth promoter site. Target validation is also possible via DNA- microarrays, stem cells or monoclonal antibodies. Recently, stem cells are being used for target identification and drug development. The application of aptamers, i.e., nucleic acids having well defined 3-D shape is gaining importance in target validation of drugs. The application of the knowledge of 'Proteomics', Transproteomics and 'Genomics' together with the array approach is likely to decide the future of drug development.
机译:迄今为止使用的常规药物是有机小分子,旨在降解病蛋白,这是病原体在细胞水平上干扰的结果。这些药物在细胞中的高浓度对于获得理想的治疗效果是必需的。这加剧了毒性问题。自从人类基因组解体以来,人们就试图将药物选择性地靶向受影响的部位,以提高其效率并同时降低其副作用。 “蛋白质组学”和“基因组学”已成为新兴学科。现在可以通过鉴定“正确的基因”,“正确的途径”和“正确的靶标”来实现任何配体(药物)的“目标识别”,并且可以开发出“正确的患者”的“正确的药物”。基因组学信息决定了目标验证,实际上是特定目标与疾病过程之间的关联。可以在DNA级别使用基因敲除策略和三链体形成,在RNA级别使用RNA干扰(RNAi),在m-RNA级别使用反义概念,在核酶级别或任何其他中间生长启动子位点上实现靶标验证。还可以通过DNA微阵列,干细胞或单克隆抗体进行靶标验证。最近,干细胞被用于目标识别和药物开发。适体即具有明确定义的3-D形状的核酸的应用在药物的靶标验证中变得越来越重要。将“蛋白质组学”,“蛋白质组学”和“基因组学”知识与阵列方法一起应用可能会决定药物开发的未来。

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