Aav vectors, aav vector compositions, and uses thereof

A combination of AAV vectors carrying Magnet photosensitive protein and Dre recombinase was used to achieve precise gene therapy for retinal damage under light-controlled conditions, solving the problem of efficient expression and diffusion of β-catenin in retinal damage and promoting retinal repair.

CN122168684APending Publication Date: 2026-06-09ACADEMY OF MILITARY MEDICAL SCIENCES
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
ACADEMY OF MILITARY MEDICAL SCIENCES
Filing Date
2024-12-06
Publication Date
2026-06-09

AI Technical Summary

Technical Problem

Current technologies lack effective methods to restore retinal damage, particularly through gene therapy to enable β-catenin to be efficiently expressed and spread in retinal MG cells, promoting its entry into the cell cycle to repair retinal damage.

Method used

By using an AAV vector carrying Magnet photosensitive protein and a segmented Dre recombinase sequence, and an AAV vector containing a Rox sequence, a catalytically active Dre recombinase is formed under light-controlled conditions, which precisely controls the expression of target protein genes and achieves stable and efficient expression of specific genes.

Benefits of technology

It enables precise control of target protein expression within specific time and space, improves gene editing efficiency, and is widely used in the development of gene editing technology and gene therapy drugs, especially in the treatment of retinal damage.

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Abstract

This invention discloses a first adeno-associated virus (AAV) vector, a composition comprising a first AAV vector and a second AAV vector, and the application thereof. The first AAV vector contains a nucleotide sequence encoding a fusion protein encoding a Magnet photosensitizing protein and a Dre recombinase fragment, wherein the Magnet photosensitizing protein comprises nMag and pMag, and the Dre recombinase fragment comprises Dre… 1‑246 Fragments and Dre 247‑342 The Magnet photosensitive protein and the Dre recombinase fragment are linked by linkers L4 and T2A. The structure of the fusion protein is shown below: Dre 1‑246 Fragment - L4 - nMag - T2A - pMag - L4 - Dre 247‑342 The first fragment; the second AAV vector carries the Rox sequence and the target protein gene sequence. The AAV vector expression system of this invention is stable and has high expression efficiency. By controlling light conditions, it can achieve precise control of target protein expression within specific time and space, and is widely used in gene editing technology development, gene function research, and the development of gene therapy drugs.
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