SnRNA targeting USH2A pre-mRNA and application thereof
AssigneeGuangzhou Reforgene Medicine Co., Ltd.
InventorsJunbin Liang, Jiayu Ou, Hui Xu, Simiao Lin
Life of this patent
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Abstract
Provided is snRNA which targets USH2A pre-mRNA. The recognition domain of the snRNA is the reverse complement of a USH2A pre-mRNA sequence. The snRNA binds to the USH2A pre-mRNA to splice and jump exon 13. The snRNA promotes a higher exon 13 skipping efficiency than AON.
Claims11
An snRNA that targets an USH2A pre-mRNA to induce splicing skipping of Exon 13, wherein the snRNA comprises a recognition domain that is reversely complementary to the USH2A pre-mRNA sequence, a smOPT sequence shown in SEQ ID NO:
- 31, and a U7-snRNA scaffold sequence, wherein the recognition domain of the snRNA is selected from the following sequences:
- SEQ ID NOs:
- 11, 15, and 17.
A nucleic acid, wherein the nucleic acid comprises a nucleotide sequence encoding an snRNA;
- wherein the snRNA comprises a recognition domain that is reversely complementary to an USH2A pre-mRNA sequence, a smOPT sequence shown in SEQ ID NO:
- 31, and a U7-snRNA scaffold sequence, wherein the recognition domain of the snRNA is selected from the following sequences:
- SEQ ID NOs:
- 11, 15 and 17, and wherein the snRNA binds to the USH2A pre-mRNA to induce splicing skipping of Exon 13.
A method for preventing or treating retinitis Pigmentosa associated with Usher syndrome type 2 in a subject in need thereof, wherein the method comprises administering an effective amount of one or more selected from an snRNA, or a vector encoding the snRNA to the subject;
- optionally wherein the snRNA or the vector encoding the snRNA is in a viral particle, a cell, or a pharmaceutical composition;
- wherein the snRNA comprises a recognition domain that is reversely complementary to an USH2A pre-mRNA sequence, a smOPT sequence shown in SEQ ID NO:
- 31, and a U7-snRNA scaffold sequence, wherein the recognition domain of the snRNA is selected from the following sequences:
- SEQ ID NOs:
- 11, 15 and 17, and wherein the snRNA binds to the USH2A pre-mRNA to induce splicing skipping of Exon 13.
Dependencies are inferred from the claim text.