期刊
MOLECULAR THERAPY-NUCLEIC ACIDS
卷 33, 期 -, 页码 367-375出版社
CELL PRESS
DOI: 10.1016/j.omtn.2023.07.014.
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Trans-acting hammerhead ribozyme inherits the advantages of being the smallest and best-characterized RNA-cleaving ribozyme, offering high modularity and the ability to cleave any desired sequence without the aid of any protein, as long as the target sequence contains a cleavage site. Precise control over the trans-acting hammerhead ribozyme has been achieved through intracellular selection of hammerhead aptazyme, leading to the development of new cis-acting hammerhead aptazymes that can efficiently knock down targeted genes in eukaryotic cells through induction by theophylline. The best aptazyme, T195, exhibits a ligand-dependent and dose-dependent response to theophylline, with enhanced cleavage efficiency when multiplex aptazymes are incorporated.
Trans-acting hammerhead ribozyme inherits the advantages of being the smallest and best-characterized RNA-cleaving ribozyme, offering high modularity and the ability to cleave any desired sequence without the aid of any protein, as long as the target sequence contains a cleavage site. However, achieving precise control over the trans-acting hammerhead ribozyme would enable safer and more accurate regulation of gene expression. Herein, we described an intracellular selection of hammerhead aptazyme that contains a theophylline aptamer on stem II based on toxin protein IbsC. Based on the intracellular selection, we obtained three new cis-acting hammerhead aptazymes. Moreover, the corresponding trans-acting aptazymes could be efficiently induced by theophylline to knock down different targeted genes in eukaryotic cells. Notably, the best one, T195, exhibited a ligand-dependent and dosedependent response to theophylline, and the cleavage efficiency could be enhanced by incorporating multiplex aptazymes.
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