Antisense RNA editing oligonucleotides including cytidine analogs

By using cytidine analogs such as pseudoisocytidine (piC) and Benna base Z (dZ) in RNA editing oligonucleotides (AON), the problem of low deamination efficiency of target RNA-specific adenosine in the prior art is solved, and efficient and specific RNA editing is achieved, enhancing the application potential of the technology in human therapy.

CN113994000BActive Publication Date: 2025-03-25PROQR THERAPEUTICS NV +1
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Patent Information

Application Number
CN202080042924.1
Authority / Receiving Office
CN · China
Patent Type
Patents(China)
Current Assignee / Owner
Priority Date
2019-06-13
Filing Date
2020-06-12
Publication Date
2025-03-25
Estimated Expiration
2040-06-12

AI Technical Summary

Technical Problem

Existing RNA editing technologies are difficult to achieve efficient deamination of specific adenosines in target RNA, and gene modification and expression of exogenous ADAR proteins are often required, which limits its application in human therapy.

Method used

Antisense oligonucleotides (AON) containing cytidine analogs, such as pseudoisocytidine (piC) and Benna base Z (dZ) act as H-bond donors at the N3 site, which can effectively recruit endogenous ADAR enzymes to achieve deaminization of specific adenosines in the target RNA.

Benefits of technology

By using cytidine analog modified AON, the efficiency and specificity of target RNA editing is significantly improved, confounding editing is avoided, and the expression of exogenous ADAR protein is not required, enhancing the potential of the technology in human therapy.

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Abstract

The present invention relates to single-stranded RNA editing antisense oligonucleotides (AONs) for binding to a target RNA molecule, deaminating at least one target adenosine present in the target RNA molecule and recruiting the ADAR2 enzyme in a cell, preferably a human cell, to deaminize at least one target adenosine in the target RNA molecule. The AON according to the present invention includes a cytidine analog at a position relative to the target adenosine, wherein the cytidine analog acts as an H-bond donor at the N3 site for more efficient RNA editing.
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