Application of microglial cell TAK1 gene as target spot in treatment of retinal neovascular diseases

By knocking out the TAK1 gene in microglia and using a recombinant lentiviral vector carrying sgRNA and a microglia-specific promoter, the lack of macro-regulation in the treatment of existing retinal neovascularization diseases has been solved, achieving specific inhibition and therapeutic effects on retinal neovascularization.

CN121102477APending Publication Date: 2025-12-12THE FIRST AFFILIATED HOSPITAL OF JINAN UNIV
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Patent Information

Application Number
CN202511256379.7
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Filing Date
2025-09-03
Publication Date
2025-12-12

AI Technical Summary

Technical Problem

Current treatments for retinal neovascularization lack macroscopic regulation of the inflammatory microenvironment of the retina and may affect normal angiogenesis and development. Existing gene therapies mainly target endothelial cells and are not very effective.

Method used

By knocking out the TAK1 gene in microglia, a recombinant lentiviral vector carrying sgRNA and a microglia-specific promoter is used to specifically inhibit pathological retinal angiogenesis. CRISPR-Cas9 technology is used to efficiently knock out the TAK1 gene in microglia, avoiding interference with normal angiogenesis.

Benefits of technology

It effectively inhibits the formation of pathological retinal neovascularization and treats retinal neovascular diseases such as diabetic retinopathy, retinopathy of prematurity, retinal vein occlusion, and familial exudative vitreoretinopathy, without affecting normal angiogenesis.

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Abstract

The invention relates to application of a microglial cell TAK1 gene as a target spot in treatment of retinal neovascular diseases. The invention finds that by specifically knocking out the microglial cell TAK1 gene, the number of neovascular clusters under pathological conditions can be effectively reduced, migration and proliferation of retinal tissue neovascular endothelial cells are inhibited, pathological retinal neovascularization is effectively inhibited, but the physiological angiogenesis capability of human retinal microvascular endothelial cells is not influenced. Therefore, retina neovascular diseases including diabetic retinopathy, premature infant retinopathy, retinal vein occlusion, familial exudative vitreoretinopathy and the like can be effectively treated by specifically knocking out the microglial cell TAK1 gene. The invention provides a new target and a new idea for the treatment of retinal neovascular diseases.
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