Vectors and Gene Delivery to the Retina
Open accessAbstract
One of the great advantages of the retina as a target tissue for gene delivery is the wide array of genetic tools that have been developed in the past decade. This includes a variety of vectors for therapeutic gene delivery to most types of retinal neurons and glia, as well as cell type–specific promoters for restricted gene expression in distinct neuronal subtypes. Within the scope of neuroscience applications and for gene therapy, it is now routine to express reporter genes, replacement genes, neuronal activity indicators, and microbial opsins in specific neuronal types in the mouse retina. However, there are considerable anatomical, physiological, immunological, and behavioral differences between the mouse and the human that limit the usefulness of these tools in humans and nonhuman primates. Several advances are now being made toward the goal of applying viral targeting tools to understand the primate retina. Here, we describe these advances, consider their potential to advance our understanding of the primate retina, and describe what will be needed to move forward.
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Adenoviral vectors tend to be rapidly cleared due to the presence of neutralizing antibodies from pre-existing immunity.
“However, these vectors tend to be rapidly cleared due to the presence of neutralizing antibodies from pre-existing immunity.4,29,54–56”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
Retroviruses and lentiviruses carry risks for insertional mutagenesis and germline transmission and may elicit more inflammatory responses than AAVs.
“However, they carry risks for insertional mutagenesis and germline transmission and may elicit more inflammatory responses than AAVs.54,56,73”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
Adenoviral vectors show good safety when administered to target tissues, do not integrate in the recipient genome, successfully transduce retinal cells, and can carry genes of around 30 kb long.
“These viruses show good safety when administered to target tissues, do not integrate in the recipient genome, successfully transduce retinal cells, and can carry genes of around 30 kb long.4,29,54–56”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
RPE cells can be targeted using AAV1, AAV4, AAV6, or the engineered AAV2-7m8 vector; photoreceptors and Müller glial cells are targeted by serotypes AAV2, AAV5, AAV6, AAV7, AAV8, AAV9, AAV2-7m8, and AAV8BP2.
“RPE cells can be targeted using AAV1, AAV4, or AAV6 vectors or with the engineered AAV2-7m8 vector, and photoreceptors and Müller glial cells are targeted by serotypes AAV2, AAV5, AAV6, AAV7, AAV8, and AAV9 as well as engineered AAV2-7m8 and AAV8BP2 vectors.56,112–116”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
Adenoviruses and AAVs are known to have tissue-specific tropisms that make certain serotypes attractive for development of retinal gene therapy products.
“Adenoviruses and AAVs are known to have tissue-specific tropisms that make certain serotypes attractive for development of retinal gene therapy products.14,29,56”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
It is generally preferred to use a delivery route that brings the therapy as close to the target tissue as possible, such as subretinal injections for outer retina targets and intravitreal injections for inner retina targets.
“although generally it is preferred to use a route that will bring the therapy as close to the target tissue as possible, such as subretinal injections for outer retina targets and intravitreal injections for inner retina targets.56”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
Recombinant vectors AAV2.GL and AAV2.NN improve retinal delivery of a gene therapy product after intravitreal injection.
“However, development of recombinant vectors AAV2.GL and AAV2.NN improve the retinal delivery of a gene therapy product after intravitreal injection.56,66”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [56]
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References 109 · Cited by 67
- Authors
- Planul, A., & Dalkara, D.
- Affiliations
- Centre National de la Recherche Scientifique (FR)
- ORCID
- Dalkara, Deniz
- Venue
- Annual Review of Vision Science
- Publisher
- Annual Reviews
- Published
- 2017-09-15
- Volume
- 3
- Issue
- 1
- Pages
- 121-140
- ISSN
- 2374-4642 (ISSN-L)
- 2374-4650
- OpenAlex
- W2589218963
- Cited by
- 67
- References
- 109
- Citations by year
- 2026: 3 · 2025: 8 · 2024: 11 · 2023: 5 · 2022: 9 · 2021: 11 · 2020: 6 · 2019: 7 · 2018: 6 · 2017: 1
- FWCI
- 0.51
- Type
- journal article
- Language
- en
- Open access
- Licence
- other-oa
- Locations
- Annual Review of Vision Science (publishedVersion)
- PubMed (publishedVersion)
- HAL (submittedVersion, other-oa)
- 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 delivery, viral vectors, retinal gene therapy, cell type-specific promoters, retinal neurons, retinal glia, reporter genes, microbial opsins, primate retina
- MeSH terms
- Adenoviridae, Animals, Genetic Vectors, Humans, Primates, Promoter Regions, Genetic, Retinal Diseases, Genetic Therapy, Lentivirus, Gene Transfer Techniques, Nanoparticles, Optogenetics
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