Abstract: Provided is a method for editing a target RNA at a target residue position of a host cell. The method comprises introducing ADAR-recruiting RNA (arRNA) or a construct encoding the arRNA into a host cell, wherein the arRNA comprises a complementary RNA sequence that hybridizes to a target RNA; the target residue is located in a three-base motif comprising a 5? nearest-neighbor residue (upstream residue) of the target residue in the target RNA, the target residue, and a 3? nearest-neighbor residue (downstream residue) of the target residue in the target RNA, wherein the three-base motif is not UAG, and the complementary RNA sequence comprises a mismatch directly opposite to the upstream residue or the downstream residue in the target RNA. Further provided are arRNA for the method, an RNA obtained by the method, a host cell comprising the RNA, and use of the method in the treatment of a disease.

Abstract: Disclosed are a LEAPER technique-based nucleic acid drug and a method for treating diseases such as Hurler syndrome by means of using the nucleic acid drug to target and edit RNA. The method comprises: performing an editing from adenosine base to hypoxanthine base on RNA by using the nucleic acid drug to precisely repair the pathogenic G>A mutation site of, for example, Hurler syndrome; thereby recovering the normal in vivo expression of a protein encoded by RNA, such as IDUA.

Abstract: The present application provides methods for editing RNA by introducing a deaminase-recruiting RNA in a host cell for deamination of an adenosine in a target RNA. The present application further provides deaminase-recruiting RNAs used in the RNA editing methods and compositions comprising the same.

Abstract: Provided is a method for targeted editing of target RNA containing a G to A mutation in a USH2A gene transcript based on LEAPER technology, comprising: introducing a construct of an adenosine deaminase recruiting RNA (arRNA) for editing the target RNA or a construct encoding said arRNA into a cell, wherein the arRNA comprises a complementary RNA sequence that hybridizes to the target RNA, and wherein the arRNA is capable of recruiting adenosine deaminase acting on RNA (ADAR), so that the target adenosine in the target RNA is deaminated, thereby performing in vivo editing of the base from A to I on RNA safely and effectively, repairing a pathogenic mutation site, and achieving the purpose of treating disease such as Usher syndrome.

Abstract: The present application relates to an LEAPER-based method for targeted editing of RNA, which comprises: safely and effectively performing adenosine-to-inosine RNA editing (A-to-I RNA editing) by LEAPER in vivo, thereby the pathogenic mutation sites can be accurately repaired, and all diseases caused by G>A mutations, such as MPS IH, can be treated.

Abstract: Provided is a method for deaminating targeted cytosine in a target RNA of a cell. The method comprises: introducing, into the cell, a modified adenosine deaminase protein or a catalytic domain thereof, or a construct expressing the modified adenosine deaminase protein or the catalytic domain thereof, and a construct comprising an arRNA oligonucleotide that recruits the modified adenosine deaminase protein or the catalytic domain thereof to the target RNA or expressing arRNA. Further provided are an engineered composition or system for RNA editing, and a use of treating a disease by using the engineered composition or system to correct a T-C mutation.

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. Further provided are deaminase-recruiting RNAs used in the RNA editing methods and compositions comprising the same.

Abstract: The present application provides methods for editing RNA by introducing a deaminase-recruiting RNA in a host cell for deamination of an adenosine in a target RNA. The present application further provides deaminase-recruiting RNAs used in the RNA editing methods and compositions comprising the same.