Molecular switch for inducible gene expression

WO2025097124A1PCT designated stage expired Publication Date: 2025-05-08THE BRIGHAM & WOMEN S HOSPITAL INC +1
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Patent Information

Application Number
PCT/US2024/054393
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-11-04
Filing Date
2024-11-04
Publication Date
2025-05-08

AI Technical Summary

Technical Problem

Existing gene switches for regulating gene expression are often large in size, making them difficult to incorporate into gene therapy vectors like AAV, and they introduce foreign proteins that can cause immunogenicity.

Method used

Development of small (9-nucleotide) termination-readthrough-based gene switches, such as TREAD and TREAD-dimmer, which use a TRSTOP sequence to minimize transgene expression and can be controlled by chemical inducers like gentamicin, without introducing foreign proteins.

Benefits of technology

These gene switches allow for precise control of transgene expression, are compatible with AAV vectors, and reduce the risk of immunogenicity, enhancing the safety and efficacy of gene therapy.

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Abstract

Described herein are termination-readthrough-based gene switches that are small (only 9 nucleotides) and do not introduce any foreign protein into the target cell, and that can be used to alter expression levels of transgenes in a cell.
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Citation Information

Patent Citations

  • AAV delivery of nucleobase editors

    US20220249697A1

  • Nucleic acid construct

    WO2023139360A1