Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

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...

The Retinoblastoma Gene

The Retinoblastoma Gene

Tumor suppressor genes are normal genes that can slow down cell division, repair DNA mistakes, or program the cells for apoptosis in case of irreparable damage. Hence, they play an essential role in preventing the proliferation of damaged cells.
The first-ever tumor suppressor gene called Rb was identified in retinoblastoma - a rare eye tumor in children. In inherited forms of the disease, a child inherits one defective copy of the Rb gene, which predisposes them to retinoblastoma. However,...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Optimizing Patient Selection for Panel-Based Molecular Diagnosis in Inherited Retinal Disease: A Health Economics-Oriented Taiwanese Cohort Study.

Ophthalmic epidemiology·2026

Same author

GLP-1 Receptor Agonists and Age-related Macular Degeneration Risk in Diabetes or Non-diabetic Obesity: A Retrospective Cohort Study.

The American journal of medicine·2026

Same author

<i>Nostoc commune</i> Extract Attenuates Oxidative Stress and Neuroinflammation in Ischemic Optic Neuropathy Through PI3K/AKT/mTOR Signaling.

Antioxidants (Basel, Switzerland)·2026

Same author

Adaptive immune crosstalk and complement dysregulation in the aging retina.

Tzu chi medical journal·2026

Same author

Janus Kinase inhibitors associated with reduced anterior uveitis risk in autoimmune diseases.

Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie·2026

Same author

Glycosylation-driven necroptosis in retinal degeneration: dual rescue by AAV8 gene therapy and RIPK1 inhibition.

Cell death discovery·2026

Search research articles

Related Experiment Video

Updated: Aug 15, 2025

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 in hereditary retinal dystrophy.

Jia-Ying Chien¹, Shun-Ping Huang^1,2,3

¹Institute of Medical Science, Tzu Chi University, Hualien, Taiwan.

Tzu Chi Medical Journal

|December 29, 2022

Summary

Gene therapy offers new hope for hereditary retinal dystrophies (HRDs). This review explores four innovative gene-targeting approaches to potentially slow or reverse vision loss in patients with these genetic retinal disorders.

Keywords:

Antisense oligonucleotides Clustered Regularly Interspaced Short Palindromic Repeat/cas system Gene therapy Optogenetics Retinal dystrophy

More Related Videos

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

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

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

Published on: November 25, 2012

Related Experiment Videos

Last Updated: Aug 15, 2025

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

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

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

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

Published on: November 25, 2012

Area of Science:

Ophthalmology
Genetics
Molecular Biology

Background:

Hereditary retinal dystrophies (HRDs) are a diverse group of genetic disorders causing progressive vision loss.
HRDs present significant challenges due to genetic and phenotypic heterogeneity, often leading to legal or complete blindness.

Purpose of the Study:

To provide a comprehensive overview of current gene therapy strategies for HRDs.
To highlight four key gene-targeted therapeutic approaches with potential for vision restoration.

Main Methods:

Review of recent advancements in gene therapy for HRDs.
Focus on four distinct therapeutic modalities: gene delivery, RNA-based therapy, genome editing, and optogenetics.

Main Results:

Gene augmentation therapy has shown promise, with the first FDA approval for Leber's congenital amaurosis (LCA).
Multiple clinical trials are actively investigating various gene therapy techniques for different HRDs.

Conclusions:

Gene therapy represents a rapidly advancing field for treating HRDs.
The explored approaches hold significant potential to slow or reverse retinal degeneration and improve patient outcomes.