Nucleotide and use thereof
AssigneeGuangzhou Reforgene Medicine Co., Ltd.
InventorsJunbin Liang, Jiayu Ou, Hui Xu, Simiao Lin
Life of this patent
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Abstract
Provided are a nucleotide and a use thereof. Specifically provided is a nucleic acid molecule that specifically binds to exon 13 of USH2A pre-mRNA. Also provided is a preparation method for and use of the nucleic acid molecule. The nucleic acid molecule has the ability to specifically bind to sequence 3′ of exon 13 of USH2A pre-mRNA or a fragment thereof, can perform targeted interference on Pre-mRNA splicing, and can increase the proportion of single skipping of exon 13 of USH2A pre-mRNA, so that safety can be ensured while efficiency is significantly improved.
Claims20
A nucleic acid molecule having the ability to specifically bind to the 3′ sequence of exon 13 of USH2A pre-mRNA or a fragment thereof, wherein the genomic location corresponding to the 3′ sequence of exon 13 of USH2A pre-mRNA is Chr1:
- 216246563-216246753;
- or, the nucleic acid molecule has the ability to specifically bind to the sequence of SEQ ID NO:
- 1 or a fragment thereof.
Dependencies are inferred from the claim text.