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Heterobifunctional small molecules to modulate RNA function
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DOI：10.1016/j.tips.2024.03.006.png)
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RNA has diverse cellular functionality, including regulating gene expression, protein translation, and cellular response to stimuli, due to its intricate structures. Over the past decade, small molecules have been discovered that target functional structures within cellular RNAs and modulate their function. Simple binding, however, is often insufficient, resulting in low or even no biological activity. To overcome this challenge, heterobifunctional compounds have been developed that can covalently bind to the RNA target, alter RNA sequence, or induce its cleavage. Herein, we review the recent progress in the field of RNA-targeted heterobifunctional compounds using representative case studies. We identify critical gaps and limitations and propose a strategic pathway for future developments of RNA-targeted molecules with augmented functionalities.
Keyword:
SECONDARY STRUCTURE PREDICTION
SEQUENCE-BASED DESIGN
NONCODING RNAS
MYOTONIC-DYSTROPHY
INTERFERON ACTION
RATIONAL DESIGN
GENOME SEQUENCE
WEB SERVER
CLEAVAGE
DISCOVERY
期刊
IF:
19.9
论文数:
3.3K
被引数:
1.5W
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