Not so pseudo anymore: pseudogenes as therapeutic targets.
Not so pseudo anymore: pseudogenes as therapeutic targets.
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DOI：
10.2217/pgs.13.172
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发表时间：
2013-12
期刊：
影响因子：
2.1
通讯作者：
Morris KV
中科院分区：
文献类型：
--
作者：
Roberts TC;Morris KV
假基因是由失活突变或调控序列丢失产生的垃圾DNA基因残留物，通常发生在基因复制或逆转录转座事件之后。这些假基因以前被认为是来自曾经编码基因的分子化石。在许多情况下，假基因并不赋予宿主生物可观察到的选择优势，并且可能处于从基因组中移除的路径上。然而，假基因也可以作为新功能的原材料，特别是在基因表达调控方面。许多假基因作为非编码RNA基因复活，它们在基于RNA的基因调控回路中发挥作用。因此，功能性假基因可以简单地被认为是“基因”。在这里，我们讨论了这些假基因衍生的RNA作为人类疾病背景下基因表达调节因子的作用。特别是，我们认为通过使用反义寡核苷酸，siRNA，适体或经典的基因治疗方法作为新的药理学策略的假基因转录本的操作。
Pseudogenes are junk DNA gene remnants generated by inactivating mutations or the loss of regulatory sequences, often following gene duplication or retrotransposition events. These pseudogenes have previously been considered to be molecular fossils derived from once-coding genes. In many cases, pseudogenes confer no observable selective advantage to the host organism and may be on a path towards removal from the genome. However, pseudogenes can also serve as raw material for the exaptation of novel functions, particularly in relation to the regulation of gene expression. Many pseudogenes are resurrected as noncoding RNA genes, which function in RNA-based gene regulatory circuits. As such, functional pseudogenes might simply be considered as ‘genes’. Here, we discuss the role of these pseudogene-derived RNAs as regulators of gene expression in the context of human disease. In particular, we consider the manipulation of pseudogene transcripts through the use of antisense oligonucleotides, siRNAs, aptamers or classical gene therapy approaches as novel pharmacological strategies.