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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
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Retina-directed gene therapy: Achievements and remaining challenges
Josef Biber1, Catharina Gandor2, Elvir Becirovic2
1Department of Ophthalmology, LMU University Hospital, LMU Munich, 80336 Munich, Germany.
Pharmacology & Therapeutics
|April 23, 2025
Summary
Gene therapy using adeno-associated virus (AAV) vectors shows promise for inherited retinal diseases. Ongoing research aims to overcome challenges and expand AAV gene therapy to acquired retinopathies like AMD and DR.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Gene therapy offers novel treatments for genetic and acquired diseases by modifying gene function.
- Adeno-associated virus (AAV) vectors are a versatile and safe tool for gene delivery to retinal cells.
- Voretigene neparvovec (Luxturna) is the first approved gene therapy for RPE65-associated retinopathy, marking a milestone.
Purpose of the Study:
- To review the current status of gene therapy for retinal diseases.
- To highlight the challenges and limitations of existing AAV-based gene therapy approaches.
- To discuss future directions and innovative strategies in retinal gene therapy.
Main Methods:
- Review of current literature on AAV-based gene therapy for retinal diseases.
- Analysis of approved gene therapies and ongoing clinical trials.
- Exploration of novel strategies such as AAV capsid engineering and CRISPR/Cas genome editing.
Main Results:
- AAV gene delivery is effective and safe for retinal cells, as demonstrated by Luxturna.
- Challenges persist in treating a broader range of inherited and acquired retinopathies.
- Innovative strategies are being developed to enhance efficacy and safety.
Conclusions:
- Retinal gene therapy, particularly using AAV vectors, holds significant therapeutic potential.
- Overcoming current challenges requires continued innovation in vector design and gene editing technologies.
- Future research will likely expand gene therapy applications to complex acquired retinal diseases like age-related macular degeneration and diabetic retinopathy.
