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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...

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Related Experiment Video

Updated: Jun 13, 2026

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

Nanoparticles for retinal gene therapy.

Shannon M Conley¹, Muna I Naash

¹University of Oklahoma Health Sciences Center, Department of Cell Biology, BMSB 781, 940 Stanton L. Young Blvd, Oklahoma City, OK 73104, USA.

Progress in Retinal and Eye Research

|May 11, 2010

Summary

This summary is machine-generated.

Non-viral gene therapies, particularly polymer-compacted DNA nanoparticles, show promise for treating inherited retinal diseases. Future development requires practical strategies to address the genetic complexity of these rare ocular conditions.

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Area of Science:

Ophthalmology
Gene Therapy
Nanotechnology

Background:

Ocular gene therapy is an advancing field with viral vectors in clinical trials for Leber's congenital amaurosis (LCA).
Non-viral gene therapy approaches are gaining traction for various ophthalmic conditions.
Polymer-compacted DNA nanoparticles represent a promising non-viral vector technology.

Purpose of the Study:

To review the progress and future potential of nanoparticles in ocular gene therapy.
To discuss the requirements for developing clinically viable non-viral ocular therapeutics.
To highlight the challenges posed by the genetic heterogeneity of inherited retinal pathologies.

Main Methods:

Review of current literature on ocular gene therapy.
Focus on non-viral vector technologies, specifically polymer-compacted DNA nanoparticles.
Analysis of treatment strategies for inherited retinal diseases.

Main Results:

Viral gene therapies are progressing in clinical trials for LCA.
Non-viral gene therapy, especially using DNA nanoparticles, is a key area of development.
The genetic diversity of inherited retinal diseases presents a significant challenge for therapeutic development.

Conclusions:

Non-viral gene therapies, particularly DNA nanoparticles, hold significant promise for ocular applications.
Translating these technologies into viable treatments requires addressing practical therapeutic strategies.
The rarity and genetic complexity of inherited retinal diseases necessitate innovative approaches for effective gene therapy.