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Advances and Challenges in Gene Therapy for Inherited Retinal Dystrophies: A Comprehensive Review
Raina Jain1, Sachin Daigavane1
1Ophthalmology, Jawaharlal Nehru Medical College, Datta Meghe Institute of Higher Education & Research, Wardha, IND.
Cureus
|October 23, 2024
Summary
Gene therapy offers new hope for inherited retinal dystrophies (IRDs), a group of genetic disorders causing vision loss. Research is advancing treatments to restore sight and halt disease progression in IRDs.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Inherited retinal dystrophies (IRDs) are genetic disorders causing progressive vision loss.
- Degeneration of retinal photoreceptors is the primary pathology in IRDs.
- Gene therapy presents a potential therapeutic strategy for IRDs.
Purpose of the Study:
- To provide a comprehensive review of gene therapy innovations for IRDs.
- To discuss mechanisms, recent advancements, and challenges in gene therapy for IRDs.
- To explore future directions and enhance therapeutic efficacy for retinal diseases.
Main Methods:
- Review of current literature on gene therapy for IRDs.
- Discussion of viral and non-viral vector systems.
- Examination of gene editing technologies like CRISPR/Cas9.
Main Results:
- Luxturna approval for RPE65-mediated retinal dystrophy marks a significant milestone.
- Advancements in gene therapy vectors and delivery methods are ongoing.
- Challenges include vector delivery, long-term safety, and patient response variability.
Conclusions:
- Gene therapy holds significant promise for treating IRDs and restoring vision.
- Further research is crucial to overcome existing challenges and improve treatment outcomes.
- Innovations in gene therapy could transform the management of retinal diseases.
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