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Targeted RNA editing by leveraging endogenous adar using engineered rnas

a technology of rna editing and endogenous adar, which is applied in the direction of dsdna viruses, viruses/bacteriophages, genetic material ingredients, etc., can solve the problems of ectopic expression of proteins or their domains of non-human origin that have potential risk of eliciting immunogenicity, and the current adar-mediated rna editing technology has certain limitations

Pending Publication Date: 2022-08-04
PEKING UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The patent describes methods for editing target RNA in a host cell using deaminase-recruiting RNAs (dRNAs) that are designed to recruit adenosine deaminase acting on RNA (ADAR). The dRNAs can be introduced into the host cell and target specific RNA sequences using complementary sequences. The methods can be used to treat or prevent diseases or conditions associated with a specific RNA target. The patent also provides constructs and kits for using the dRNAs for RNA editing.

Problems solved by technology

However, currently available ADAR-mediated RNA editing technologies have certain limitations.
For example, the most effective in vivo delivery for gene therapy is through viral vectors, but the highly desirable adeno-associated virus (AAV) vectors are limited with the cargo size (˜4.5 kb), making it challenging for accommodating both the protein and the guide RNA.
In addition, ectopic expression of proteins or their domains of non-human origin has potential risk of eliciting immunogenicity.
Moreover, pre-existing adaptive immunity and p53-mediated DNA damage response may compromise the efficacy of the therapeutic protein, such as Cas9.

Method used

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  • Targeted RNA editing by leveraging endogenous adar using engineered rnas
  • Targeted RNA editing by leveraging endogenous adar using engineered rnas
  • Targeted RNA editing by leveraging endogenous adar using engineered rnas

Examples

Experimental program
Comparison scheme
Effect test

embodiment 1

2. The method of embodiment 1, wherein the dRNA further comprises a 3′ ligation sequence and a 5′ ligation sequence.

embodiment 2

3. The method of embodiment 2, wherein the 3′ ligation sequence and the 5′ ligation sequence are at least partially complementary to each other.

4. The method of embodiment 2 or embodiment 3, wherein the 3′ ligation sequence and the 5′ ligation sequence are about 20 to about 75 nucleotides in length.

5. The method of any one of embodiments 1-4, wherein the dRNA is circularized by RNA ligase RtcB.

embodiment 4

6. The method of embodiment 4, wherein the RNA ligase RtcB is expressed endogenously in the host cell.

7. The method of any one of embodiments 1-6, wherein the dRNA is a circular RNA.

8. The method of any one of embodiments 1-6, wherein the dRNA is a linear RNA capable of forming a circular RNA.

9. The method of any one of embodiments 1-6, wherein the method comprises introducing a construct comprising a nucleic acid encoding the dRNA into the host cell.

10. The method of claim 9, wherein the construct further comprises a 3′ twister ribozyme sequence linked to the 3′ end of the nucleic acid encoding the dRNA and a 5′ twister ribozyme sequence linked to the 5′ end of the nucleic acid encoding the dRNA.

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Abstract

Provided are methods for editing RNA by introducing a deaminase-recruiting RNA in a host cell for deamination of an adenosine in a target RNA, deaminase-recruiting RNAs used in the RNA editing methods, compositions and kits comprising the same.

Description

CROSS REFERENCE TO RELATED APPLICATIONS[0001]This application claims priority benefit of International Patent Application No. PCT / CN2019 / 095802 filed Jul. 12, 2019, the contents of which are incorporated herein by reference in their entirety.SUBMISSION OF SEQUENCE LISTING ON ASCII TEXT FILE[0002]The content of the following submission on ASCII text file is incorporated herein by reference in its entirety: a computer readable form (CRF) of the Sequence Listing (file name: FD00254PCT-ST25.txt, date recorded: Jul. 10, 2020, size: 16 KB).FIELD[0003]The present disclosure relates generally to methods and compositions for editing RNAs using an engineered RNA capable of recruiting an adenosine deaminase to deaminate one or more adenosines in target RNAs.BACKGROUND[0004]Genome editing is a powerful tool for biomedical research and development of therapeutics for diseases. Editing technologies using engineered nucleases, such as zinc finger nucleases (ZFNs), transcription activator-like effe...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): C12N15/10A61K31/7088C12N15/113C12N15/86
CPCC12N15/102A61K31/7088C12N15/113C12N2710/16145C12Y305/04004C12N2310/11C12N2310/532C12N15/86C12N15/63C12N2320/34C12N15/11C12N2740/16043C12N2310/3519C12N9/78A61K48/00C12N2310/12C12N2750/14143
Inventor WEI, WENSHENGYI, ZONGYIQU, LIANGTIAN, FENGWANG, CHUNHUIZHU, SHIYOUZHOU, ZHUO
Owner PEKING UNIV
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