Recent innovations in cutting-edge sequencing platforms have allowed the rapid identification of genes associated with communicable, noncommunicable and rare diseases. Exploitation of this collected biological information has facilitated the development of nonviral gene therapy strategies and the design of several proteins capable of editing specific DNA sequences for disease control. Small molecule-based targeted therapeutic approaches have gained increasing attention because of their suggested clinical benefits, ease of control and lower costs. Pyrrole-imidazole polyamides (PIPs) are a major class of DNA minor groove-binding small molecules that can be predesigned to recognize specific DNA sequences. This programmability of PIPs allows the on-demand design of artificial genetic switches and fluorescent probes. In this review, we detail the progress in the development of PIP-based designer ligands and their prospects as advanced DNA-based small-molecule drugs for therapeutic gene modulation.

中文翻译：

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使用吡咯-咪唑聚酰胺进行治疗性基因调控。

前沿测序平台的最新创新已使快速鉴定与传染病，非传染病和罕见病相关的基因成为可能。对收集到的生物学信息的利用促进了非病毒基因治疗策略的发展，并促进了几种能够编辑用于疾病控制的特定DNA序列的蛋白质的设计。基于小分子的靶向治疗方法因其建议的临床益处，易于控制和较低的成本而受到越来越多的关注。吡咯-咪唑聚酰胺（PIP）是一类主要的DNA小沟结合小分子，可以预先设计以识别特定的DNA序列。PIP的这种可编程性允许按需设计人工遗传开关和荧光探针。在这篇评论中，