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首页> 外文期刊>Infectious Disorders - Drug Targets (Formerly Current Drug Targets - Infectious Disorders) >Differential Splicing, Disease and Drug Targets
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Differential Splicing, Disease and Drug Targets

机译：差异剪接，疾病和药物靶向

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摘要

Genome complexity and diversity can be due to Alternative Splicing (AS), a process by which one gene can generate multiple mRNA isoforms and then several proteins. This is part of a normal process of variation on an individual, and when it is disrupted or modified, may trigger disease. To date, there are many pathologies described due to the effects of altered splicing isoforms, and effort is focused on the description of new ones. The design of drug target has to consider splicing, as in many occasions, a drug might have effect on different isoforms, instead of on the particular one implicated in the pathology. Interestingly, the strategies used to alter splicing can be used to modify a form towards the canonical one, or towards an aberrant one, when the latter one has a beneficial effect on the individual. Here we describe differential splicing, diseases produced by alterations on the mRNA isoforms, and drugs or methods used to restore these alterations.
机译：基因组的复杂性和多样性可能归因于选择性剪接（AS），一个基因可以通过该过程生成多种mRNA亚型，然后生成多种蛋白质。这是个体正常变异过程的一部分，当它被破坏或改变时，可能引发疾病。迄今为止，由于改变的剪接同工型的影响，已经描述了许多病理学，并且努力集中于对新的同工型的描述。药物靶标的设计必须考虑剪接，因为在许多情况下，药物可能会对不同的同工型产生影响，而不是对涉及病理的特定同种型产生影响。有趣的是，当后一种形式对个体具有有益的作用时，用于改变剪接的策略可用于将一种形式改变为规范形式或异常形式。在这里，我们描述了差异剪接，由mRNA亚型改变引起的疾病以及用于恢复这些改变的药物或方法。

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