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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

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Related Experiment Video

Updated: May 7, 2025

Methodology for Biomimetic Chemical Neuromodulation of Rat Retinas with the Neurotransmitter Glutamate In Vitro

Methodology for Biomimetic Chemical Neuromodulation of Rat Retinas with the Neurotransmitter Glutamate In Vitro

Published on: December 19, 2017

[Gene therapy for visual function recovery].

Kazuhiko Namekata¹, Xiaoli Guo¹, Chikako Harada¹

¹Visual Research Project, Tokyo Metropolitan Institute of Medical Science.

Nihon Yakurigaku Zasshi. Folia Pharmacologica Japonica

|January 5, 2025

Summary

This summary is machine-generated.

Gene therapy offers a novel approach to treating glaucoma, a leading cause of blindness. This research explores using gene therapy with neurotrophic factors to protect optic nerves and potentially restore vision in glaucoma patients.

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Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

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Last Updated: May 7, 2025

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Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

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Published on: November 25, 2012

Area of Science:

Ophthalmology
Neuroscience
Genetics

Background:

Glaucoma is a leading cause of age-related blindness, characterized by neurodegeneration of the optic nerve.
Current treatments focus on lowering intraocular pressure but are insufficient for many patients, necessitating novel therapeutic strategies.
Gene therapy is an emerging field with increasing clinical applications, including for neurodegenerative diseases.

Purpose of the Study:

To investigate the potential of gene therapy for glaucoma treatment.
To develop a novel gene therapy approach utilizing neurotrophic factor signaling.
To achieve both neuroprotection and axonal regeneration for vision recovery in glaucoma.

Main Methods:

Developing and applying gene therapy vectors.
Utilizing neurotrophic factor signaling pathways.
Investigating therapeutic effects on neuroprotection and axonal regeneration.

Main Results:

The study is investigating a new gene therapy approach for glaucoma.
The research focuses on neurotrophic factor signaling for therapeutic intervention.
The aim is to demonstrate neuroprotection and promote axonal regeneration.

Conclusions:

Gene therapy holds promise as a fundamental treatment for glaucoma.
Targeting neurotrophic factor signaling could offer a dual benefit of neuroprotection and vision recovery.
Further development is needed to establish gene therapy for clinical glaucoma treatment.