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A composition for improving the effect of gene therapy for retinal degenerative diseases and its application

A composition and gene technology, applied in gene therapy, drug combination, cardiovascular system diseases, etc., can solve the problem of not exerting a significant therapeutic effect, and achieve the effect of improving visual electrophysiological response, improving therapeutic effect, and promoting functional recovery.

Active Publication Date: 2019-12-27
BEIJING TONGREN HOSPITAL AFFILIATED TO CAPITAL MEDICAL UNIV
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Problems solved by technology

[0005] This has also brought us some new thinking. For example, although the MerTK gene therapy for retinitis pigmentosa has achieved good therapeutic effects in animal experiments, it has not played a significant therapeutic effect in clinical trials.

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  • A composition for improving the effect of gene therapy for retinal degenerative diseases and its application
  • A composition for improving the effect of gene therapy for retinal degenerative diseases and its application

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Embodiment Construction

[0023] The genetic background of retinitis pigmentosa is relatively complex. So far, more than 50 related pathogenic genes have been discovered, and the MerTK gene replacement therapy has been transformed into clinical trials. MerTK-mediated phagocytosis is a self-clearing process for apoptotic cells and large groups of physiological cell debris. Previous studies including ours have confirmed that the mutation of this gene leads to abnormal cytoskeleton aggregation and phagocytosis dysfunction in the process of cell phagocytosis. If retinal pigment epithelial cells are involved, the outer segmental membrane of photoreceptor cells cannot be removed in time, leading to secondary apoptosis of photoreceptor cells and retinal degeneration.

[0024] Although gene therapy technology has made great breakthroughs in the clinical transformation of ophthalmology, some new thinking has also emerged. For example, MerTK gene therapy has achieved good therapeutic effects in animal experimen...

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Abstract

The invention discloses a composition for promoting a therapeutic effect of a retina degenerative disease gene and an application thereof. A research proves that the long-term C-MER proto-oncogene tyrosine kinase (MerTK) mutation can cause the serious reconstruction of a retina micro-environment, the growth arrest specific gene 6 (Gas6) necessary for MerTK exerting function is extremely reduced and the relative component of phagocytosis path is influenced, so that the joint overexpression of exogenous tyrosine kinase family receptor MerTK and ligand Gas6 is adopted for inducing retina pigment epithelial cell phagocytic ability, the retinitis pigmentosa pathology course caused by the cell phagocytic function disorder can be relieved and the theory and technical base are established for the treatment of such diseases.

Description

technical field [0001] The invention belongs to the field of biotechnology, and in particular relates to a composition for improving the effect of gene therapy for retinal degenerative diseases and an application thereof. Background technique [0002] With the gradual control of optical-related eye diseases and inflammatory eye diseases, retinal neurodegenerative diseases have become the main cause of irreversible blindness in the world today, affecting the vision health of tens of millions of people. Such as retinitis pigmentosa (Retinitis Pigmentosa, RP), age-related macular degeneration (Age-related MacularDegeneration, AMD) and glaucoma. The pathological basis of visual function damage caused by such diseases is the irreversible damage of retinal neuron cells, and there is no effective treatment for this in clinic. [0003] In recent years, the rapid progress of basic scientific research has brought new hope for the treatment of the above diseases; mainly including: gen...

Claims

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Application Information

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Patent Type & Authority Patents(China)
IPC IPC(8): A61K48/00A61P9/10
CPCA61K48/0025
Inventor 张敬学王宁利武珅
Owner BEIJING TONGREN HOSPITAL AFFILIATED TO CAPITAL MEDICAL UNIV
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