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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 inserted. The...

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 inserted. The...

iPS Cell Differentiation

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.

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Home
Gene Therapy Development For Retinal Diseases.

Home
Gene Therapy Development For Retinal Diseases.

Related Experiment Video

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Gene therapy development for retinal diseases.

Felix F L Reichel¹, Julia-Sophia Bellingrath², Peter Kiraly³

¹University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.

Handbook of Clinical Neurology

|May 30, 2026

View abstract on PubMed

Summary

This summary is machine-generated.

Gene therapy offers potential treatments for retinal diseases using gene augmentation and editing. Research focuses on delivery systems, challenges, and clinical applications for inherited retinal dystrophies.

Keywords:

Gene augmentation Gene editing Inherited retinal dystrophies (IRDs)Retinal gene therapy Viral vectors

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Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model

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Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Published on: February 4, 2021

Related Experiment Videos

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model

Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model

Published on: August 5, 2021

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Published on: February 4, 2021

Area of Science:

Ophthalmology
Genetics
Biotechnology

Background:

Inherited and acquired retinal diseases represent a significant unmet medical need.
Gene therapy is emerging as a promising therapeutic modality for vision restoration.

Purpose of the Study:

To provide a comprehensive overview of current gene therapy strategies for retinal diseases.
To discuss challenges and clinical applications of retinal gene therapy.

Main Methods:

Review of gene augmentation and gene editing techniques.
Discussion of viral and nonviral delivery systems.
Analysis of challenges including gene identification, vector optimization, and immune responses.

Main Results:

Exploration of clinical applications in inherited retinal dystrophies.

Summary of recent clinical trial outcomes and endpoints.

Identification of key considerations for successful clinical translation.

Conclusions:

Retinal gene therapy is rapidly advancing, with ongoing research addressing key challenges.
Successful clinical translation requires careful consideration of delivery, safety, and efficacy.
Gene therapy holds significant promise for treating a spectrum of retinal conditions.