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Gene therapy for visual loss: Opportunities and concerns
Jia Hui Lee1, Jiang-Hui Wang2, Jinying Chen3
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, Victoria, Australia.
Progress in Retinal and Eye Research
|September 1, 2018
Summary
Gene therapy offers a genetic cure for disabling diseases, with recent advancements like CRISPR/Cas enabling precise genome engineering for ocular conditions. This review explores current gene therapy approaches, challenges, and future prospects for eye diseases.
Area of Science:
- Ophthalmology
- Genetics
- Biotechnology
Background:
- Gene therapy has demonstrated significant therapeutic benefits and safety in clinical trials.
- Evidence suggests gene therapy can provide genetic cures for severe, disabling diseases.
- The FDA approval of a gene therapy for RPE65-associated Leber's congenital amaurosis marked a milestone.
Purpose of the Study:
- To review current gene therapy approaches for ocular diseases.
- To discuss the challenges and opportunities in the field of gene therapy for eye conditions.
- To explore the future prospects of gene therapy in ophthalmology.
Main Methods:
- Review of current literature on gene therapy for ocular diseases.
- Discussion of advancements in genome editing technologies, including CRISPR/Cas.
- Analysis of clinical trial outcomes and therapeutic benefits.
Main Results:
- Gene therapy shows promise for genetically curing certain disabling ocular diseases.
- Genome editing technologies, especially CRISPR/Cas, are advancing ocular disease management.
- Significant progress has been made in developing gene therapy for eye conditions.
Conclusions:
- Gene therapy is a rapidly advancing field with the potential to cure genetic eye diseases.
- CRISPR/Cas and other genome editing tools offer new therapeutic avenues for ophthalmology.
- Despite challenges, gene therapy holds considerable promise for the future of treating ocular diseases.
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