AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression
Open accessAbstract
There has been great progress in ocular gene therapy, but delivery of viral vectors to the retinal pigmented epithelium (RPE) and retina can be challenging. Subretinal injection, the preferred route of delivery for most applications, requires a surgical procedure that has risks. Herein we report a novel gene therapy delivery approach, suprachoroidal injection of AAV8 vectors, which is less invasive and could be done in an outpatient setting. Two weeks after suprachoroidal injection of AAV8.GFP in rats, GFP fluorescence covered 18.9% of RPE flat mounts and extended entirely around sagittal and transverse sections in RPE and photoreceptors. After 2 suprachoroidal injections of AAV8.GFP, GFP fluorescence covered 30.5% of RPE flat mounts. Similarly, widespread expression of GFP occurred in nonhuman primate and pig eyes after suprachoroidal injection of AAV8.GFP. Compared with subretinal injection in rats of RGX-314, an AAV8 vector expressing an anti-VEGF Fab, suprachoroidal injection of the same dose of RGX-314 resulted in similar expression of anti-VEGF Fab and similar suppression of VEGF-induced vascular leakage. Suprachoroidal AAV8 vector injection provides a noninvasive outpatient procedure to obtain widespread transgene expression in retina and RPE.
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Two studies involving non-human primates showed conflicting results in the efficacy of photoreceptor cell uptake via the suprachoroidal route.
“Two studies involving NHPs showed conflicting results in the efficacy of photoreceptor cell uptake.78,101”
From Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses (2022) · cites this paper as [101]
In the section on suprachoroidal AAV8.GFP injection in nonhuman primates and pigs (page not supplied), the paper reports GFP expression in retinal flat mounts from three rhesus monkeys, so it supports identifying this as one NHP study of suprachoroidal gene transfer. The monkey results do not specifically identify photoreceptor uptake, and this paper alone does not establish that two NHP studies had conflicting results. (LLM, conf=0.82)
Experimental — accuracy not yet measuredThere are inconsistent reports about the efficacy of the suprachoroidal route in infecting photoreceptors.
“There are inconsistent reports about its efficacy in infecting photoreceptors.”
From Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses (2022) · cites this paper as [101]
The paper’s comparative-studies discussion reports strong photoreceptor transduction after suprachoroidal AAV8 and AAV9 injection but poor transduction with AAV2. That supports variation in efficacy, not the claim that published reports are inconsistent; the passage describes the authors’ own serotype comparison (page not available in our copy). (LLM, conf=0.80)
Experimental — accuracy not yet measuredStudies on animals other than non-human primates showed that the suprachoroidal route can be used effectively to infect photoreceptors.
“Studies on animals other than NHPs showed that the suprachoroidal route can be used effectively to infect photoreceptors.95,101”
From Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses (2022) · cites this paper as [101]
Data in rat, pig, and NHP suggest plausibility of transduction of both RPE and PRs via suprachoroidal AAV gene delivery, although results have been inconsistent across studies.
“Data in rat, pig, and NHP suggest plausibility of transduction of both RPE and PRs via suprachoroidal AAV gene delivery, although results have been inconsistent across studies.116, 117, 118”
From Current clinical applications of in vivo gene therapy with AAVs (2021) · cites this paper as [116]
Subconjunctival injection often utilize AAV8 and AAV6 for gene delivery, and AAV8 is considered a reliable choice for suprachoroidal injections.
“Subconjunctival injection often utilize AAV8 and AAV6 for gene delivery67, and AAV8 is considered a reliable choice for suprachoroidal injections33,68,69.”
From An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species (2024) · cites this paper as [69]
Suprachoroidal injection has been successful in large-animal models and was demonstrated to be a safer approach in a phase 3 clinical trial to treat uveitis with macular edema.
“This method has been successful in large-animal models and was demonstrated to be a safer approach in a phase 3 clinical trial to treat uveitis with macular edema [20,21].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [21]
Suprachoroidal injection of an AAV-based vector in large animals achieved a wider distribution of outer retinal transduction than subretinal therapy.
“suprachoroidal injection of an AAV-based vector in large animals achieved a wider distribution of outer retinal transduction than subretinal therapy [310,311,312]”
From Bruch’s Membrane: A Key Consideration with Complement-Based Therapies for Age-Related Macular Degeneration (2023) · cites this paper as [311]
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- Authors
- Ding, K., Shen, J., Hafiz, Z., Hackett, S. F., Silva, R. L. E., Khan, M., Lorenc, V. E., Chen, D., Chadha, R., Zhang, M., Van Everen, S., Buss, N., Fiscella, M., Danos, O., & Campochiaro, P. A.
- Affiliations
- Johns Hopkins University (US)
- Regenxbio (United States) (US)
- Corresponding author
- Campochiaro, Peter A.
- ORCID
- Venue
- Journal of Clinical Investigation
- Published
- 2019-10-07
- Volume
- 129
- Issue
- 11
- Pages
- 4901-4911
- ISSN
- 0021-9738 (ISSN-L)
- 1558-8238
- PMC
- PMC6819121
- OpenAlex
- W2967160972
- Cited by
- 141
- References
- 37
- Citations by year
- 2026: 15 · 2025: 18 · 2024: 22 · 2023: 21 · 2022: 16 · 2021: 29 · 2020: 19
- FWCI
- 9.61
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Journal of Clinical Investigation (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Conicet (submittedVersion, cc-by-nc-sa)
- Funders
- REGENXBIO, Inc (None)
- Alsheler-Durell Foundation (none)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- suprachoroidal injection, AAV8 vector, ocular gene therapy, retinal pigment epithelium, transgene expression, subretinal injection, photoreceptors, non-human primates, pigs
- MeSH terms
- Dependovirus, Animals, Genetic Vectors, Macaca mulatta, Transduction, Genetic, Gene Expression, Transgenes, Green Fluorescent Proteins, Retinal Pigment Epithelium
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