Related Experiment Video
Updated: Aug 29, 2025
06:48
Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
21.5K
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases
Shun-Yun Cheng1, Claudio Punzo1,2,3
1Departments of Ophthalmology and Visual Sciences, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
Human Gene Therapy
|September 8, 2022
Summary
Gene therapy for inherited retinal diseases has advanced significantly since 2001, with successful canine trials paving the way for FDA approval and numerous human clinical trials. Future approvals of ocular gene therapies are anticipated.
Area of Science:
- Ophthalmology
- Genetics
- Biotechnology
Background:
- Gene therapy for inherited retinal diseases (IRDs) has progressed rapidly.
- A landmark canine gene therapy trial in 2001 for Leber's congenital amaurosis type 2 preceded FDA approval sixteen years later.
- This success spurred widespread development in ocular gene therapy.
Purpose of the Study:
- To review the current state of viral gene therapies targeting the retina.
- To highlight ongoing human clinical trials for inherited retinal dystrophies.
- To project the future of approved ocular gene therapies.
Main Methods:
- Review of published literature on viral gene therapy for retinal conditions.
- Analysis of ongoing and completed human clinical trials.
- Focus on therapies for inherited retinal dystrophies.
Main Results:
- Gene therapy has been applied to all classes of inherited retinal dystrophy in animal models.
- Numerous human clinical trials are currently underway.
- The field has expanded significantly since the initial canine success.
Conclusions:
- Viral gene therapy shows immense promise for treating inherited retinal dystrophies.
- Continued research and clinical trials are expected to lead to further FDA approvals.
- The next decade is likely to witness the approval of several new viral gene therapies for vision restoration.
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