ABCA4 trans-splicing molecules

By using nucleic acid trans splicing molecules and RNA exon editor technology, the retinal disease problem caused by ABCA4 gene mutation is solved, gene correction at the RNA level is achieved, and effective treatment options are provided.

CN119731328APending Publication Date: 2025-03-28SEA SQUIRT THERAPY CO
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Patent Information

Application Number
CN202380049353.8
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2023-01-04
Filing Date
2023-05-12
Publication Date
2025-03-28

AI Technical Summary

Technical Problem

The prior art is difficult to effectively correct mutations in the ABCA4 gene, resulting in retinal diseases such as progressive blindness, and a single base editing method cannot solve the problems of a large number of patients.

Method used

Using nucleic acid trans splicing molecules, the RNA exon editor is designed to trans splice into the mutation-containing ABCA4 mRNA precursor by delivering a therapeutic RNA trans splicing construct, and the mutation is corrected by self-therapeutic RNA exon editor's encoding functional amino acid sequence, replacing the exons in the endogenous ABCA4 mRNA precursor.

Benefits of technology

Correcting gene mutations at the RNA level is achieved, providing treatment intervention choices for ABCA4-related retinopathy, significantly improving the treatment choices and effects of patients.

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Abstract

Provided herein are nucleic acid trans-splice molecules (e.g., mRNA precursor trans-splice molecules (RTM); rNA exon editing molecules). Such molecules can be used in the treatment of diseases such as ABCA4-related retinal dystrophy (e.g., Strike's disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splice molecules described herein to correct mutations in ABCA4, thereby treating conditions associated with mutations in ABCA4, and the use of the nucleic acid trans-splice molecules described herein to treat conditions associated with mutations in ABCA4 and to prepare medicaments for treating conditions associated with mutations in ABCA4.
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