Noninvasive gene delivery to foveal cones for vision restoration
Open accessAbstract
Intraocular injection of adeno-associated viral (AAV) vectors has been an evident route for delivering gene drugs into the retina. However, gaps in our understanding of AAV transduction patterns within the anatomically unique environments of the subretinal and intravitreal space of the primate eye impeded the establishment of noninvasive and efficient gene delivery to foveal cones in the clinic. Here, we establish new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea with supporting studies in mouse models, human induced pluripotent stem cell–derived organoids, postmortem human retinal explants, and living macaques. We show that an AAV9 variant provides efficient foveal cone transduction when injected into the subretinal space several millimeters away from the fovea, without detaching this delicate region. An engineered AAV2 variant provides gene delivery to foveal cones with a well-tolerated dose administered intravitreally. Both delivery modalities rely on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. The model systems described here provide insight into the behavior of AAV vectors across species to obtain safety and efficacy needed for gene therapy in neurodegenerative disorders.
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rAAV2(quad Y-F) and rAAV2-7m8 also infected Müller glial cells and some rod photoreceptor cells ex vivo.
“rAAV2(quad Y-F) and rAAV2-7m8 also infected Müller glial cells and some rod photoreceptor cells ex vivo [293,294].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [293]
The Results report that AAV2-7m8 with the mCAR promoter produced expression in some rods and inner-retinal cells after intravitreal injection in mice, but they do not identify those cells as Müller glia or describe that result as ex vivo. The section on human retinal explants instead reports cone-specific expression with AAV2-7m8-PR1.7. Our copy does not report an ex vivo result for rAAV2(quad Y-F). (LLM, conf=0.84)
Experimental — accuracy not yet measuredThe mouse cone arrestin promoter (mCAR) also expressed eGFP in rods, inner nuclear layer cells, and ganglion cells.
“However, the mouse cone arrestin promoter (mCAR) also expressed eGFP in rods, inner nuclear layer cells, and ganglion cells [293].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [293]
The Results (page unavailable) report that, after intravitreal delivery in mice, mCAR drove GFP expression in rods and cells of the inner nuclear layer and ganglion cell layer. The paper identifies the last group by retinal layer, not specifically as ganglion cells, so that part of the claim is more specific than the evidence. (LLM, conf=0.84)
Experimental — accuracy not yet measuredSubretinal injection of rAAV1, rAAVrh64R1, rAAV2, rAAV2-7m8, rAAV5, rAAVrh8R, rAAV7, rAAV8, rAAV8BP2, rAAV9, rAnc80L65 with a ubiquitous promoter infects rod photoreceptors and RPE cells in nonhuman primates.
“Subretinal injection (ubiquitous promoter): rAAV1, rAAVrh64R1, rAAV2, rAAV2-7m8, rAAV5, rAAVrh8R, rAAV7, rAAV8, rAAV8BP2, rAAV9, rAnc80L65 infect rod photoreceptors and RPE cells [280,293,316-320].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [293]
The Results section reports subretinal AAV delivery in macaques, but it used AAV9-7m8 with the cone-specific PR1.7 promoter and found foveal cone transduction. It does not report that the listed vectors, delivered subretinally with a ubiquitous promoter, infect rods and RPE cells in nonhuman primates. (LLM, conf=0.88)
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- Authors
- Khabou, H., Garita-Hernandez, M., Chaffiol, A., Reichman, S., Jaillard, C., Brazhnikova, E., Bertin, S., Forster, V., Desrosiers, M., Winckler, C., Goureau, O., Picaud, S., Duebel, J., Sahel, J.-A., & Dalkara, D.
- Affiliations
- Centre National de la Recherche Scientifique (FR)
- Sorbonne Universités, UPMC Univ Paris 06, INSERM, CNRS, Institut de la Vision, Paris, France.
- ORCID
- Venue
- JCI Insight
- Published
- 2018-01-25
- Volume
- 3
- Issue
- 2
- Pages
- e96029
- ISSN
- 2379-3708 (ISSN-L)
- PMC
- PMC5821199
- OpenAlex
- W2784726823
- Cited by
- 124
- References
- 51
- Citations by year
- 2026: 6 · 2025: 11 · 2024: 9 · 2023: 11 · 2022: 19 · 2021: 21 · 2020: 19 · 2019: 18 · 2018: 8
- FWCI
- 10.10
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- JCI Insight (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- HAL (submittedVersion, other-oa)
- Funders
- Foundation Fighting Blindness (Wynn-Gund translational research award)
- AFM-Téléthon (Young Researcher PhD Fellowship)
- Marie Curie CIG (334130, RETINAL GENE THERAPY)
- INSERM (---)
- Labex-Lifesenses (ANR-10-LABX-65)
- Fondation NRJ (---)
- Agence Nationale pour la Recherche - Recherche Hospitalo-Universitaire en santé (Light4Deaf)
- ERC StG (OPTOGENRET, REGENETHER)
- 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
- adeno-associated virus vectors, AAV9, AAV2, subretinal injection, intravitreal injection, retinal gene therapy, macaque retina
- MeSH terms
- Intravital Microscopy, Dependovirus, Animals, Cell Line, Female, Fovea Centralis, Genetic Vectors, Humans, Macaca fascicularis, Male, Promoter Regions, Genetic, Transduction, Genetic, Vision Disorders, Genetic Therapy, Patch-Clamp Techniques, Transgenes, Models, Animal, Mice, Injections, Intraocular, Induced Pluripotent Stem Cells, Optogenetics
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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