Retinal gene therapy: surgical vector delivery in the translation to clinical trials
Open accessAbstract
, the heterogeneity of the disease phenotype needs to be considered. This necessitates tailored approaches such as subretinal or intravitreal injections with the aim to maximize transduction of target cell populations, while minimizing off-target effects and surgical complications. Strategic decisions on parameters of the application are crucial to obtain the best treatment outcomes and patient safety. While most of the current retinal gene therapy trials utilize a subretinal approach, a deeper understanding of the numerous factors and considerations in choosing one delivery approach over the other for various ocular pathologies could lead to an improved safety and treatment efficacy. In this review we survey different vector injection techniques and parameters applied in recent retinal (pre-)clinical trials. We explore the advantages and shortcomings of each delivery strategy in the setting of different underlying ocular pathologies and other relevant factors. We highlight the potential benefits for patient safety and efficacy in applying those considerations in the decision making process.
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Injection protocols dating to 2014 called for a 2-step procedure, which largely avoids causing a break in Bruch's membrane but does not completely eliminate the risk.
“Injection protocols dating to 2014 (30, 31) called for a 2-step procedure, which largely avoids causing a break in Bruch's membrane but does not completely eliminate the risk (20, 32).”
From Subretinal therapy: technological solutions to surgical and immunological challenges (2022) · cites this paper as [32]
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References 70 · Cited by 110
- Authors
- Ochakovski, G. A., Bartz-Schmidt, K. U., & Fischer, M. D.
- Affiliations
- Universitätsklinikum Tübingen (DE)
- University of Oxford (GB)
- Corresponding author
- Fischer, M. Dominik
- Venue
- Frontiers in Neuroscience
- Publisher
- Frontiers Media SA
- Published
- 2017-04-03
- Volume
- 11
- Pages
- 174-174
- ISSN
- 1662-453X (ISSN-L)
- PMC
- PMC5376580
- OpenAlex
- W2599053860
- Cited by
- 110
- References
- 70
- Citations by year
- 2026: 7 · 2025: 10 · 2024: 8 · 2023: 10 · 2022: 12 · 2021: 21 · 2020: 13 · 2019: 10 · 2018: 15 · 2017: 3
- FWCI
- 1.05
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- cc-by
- Locations
- Frontiers in Neuroscience (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- Medical Research Council (HMRXDS0)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- retinal gene therapy, vector delivery, subretinal injection, intravitreal injection, off-target effects, patient safety, transduction efficiency, ocular pathology
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