Cited by 108Virus-based gene therapy researchRetinal Development and DisordersRNA Interference and Gene Delivery

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Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

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Mowat, F M, Gornik, K R, Dinculescu, A, Boye, S L, Hauswirth, W W, Petersen-Jones, S M, Bartoe, J T·2014·Gene Therapy

DOI 10.1038/gt.2013.64PMC PMC3880610

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Abstract

Recombinant adeno-associated viruses are important vectors for retinal gene delivery. Currently utilized vectors have relatively slow onset, and for efficient transduction it is necessary to deliver treatment subretinally, with the potential for damage to the retina. Amino-acid substitutions in the viral capsid improve efficiency in rodent eyes by evading host responses. As dogs are important large animal models for human retinitis pigmentosa, we evaluated the speed and efficiency of retinal transduction using capsid-mutant vectors injected both subretinally and intravitreally. We evaluated AAV serotypes 2 and 8 with amino-acid substitutions of surface-exposed capsid tyrosine residues. The chicken beta-actin promoter was used to drive green fluorescent protein expression. Twelve normal adult beagles were injected; four dogs received intravitreal injections and eight dogs received subretinal injections. Capsid-mutant viruses tested included AAV2(quad Y-F) (intravitreal and subretinal) and self-complementary scAAV8(Y733F) (subretinal only). Contralateral control eyes received injections of scAAV5 (subretinal) or scAAV2 (intravitreal). Subretinally delivered vectors had a faster expression onset than intravitreally delivered vectors. Subretinally delivered scAAV8(Y733F) had a faster onset of expression than scAAV5. All subretinally injected vector types transduced the outer retina with high efficiency and the inner retina with moderate efficiency. Intravitreally delivered AAV2(quad Y-F) had a marginally higher efficiency of transduction of both outer retinal and inner retinal cells than scAAV2. Because of their rapid expression onset and efficient transduction, subretinally delivered capsid-mutant AAV8 vectors may increase the efficacy of gene therapy treatment for rapid photoreceptor degenerative diseases. With further refinement, capsid-mutant AAV2 vectors show promise for retinal gene delivery from an intravitreal approach.

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Authors: Mowat, F. M., Gornik, K. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., Petersen-Jones, S. M., & Bartoe, J. T.
Affiliations: Michigan State University (US)
University of Florida Health (US)
ORCID: Mowat, F M
Gornik, K R
Dinculescu, A
Boye, S L
Hauswirth, W W
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2013-11-14
Volume: 21
Issue: 1
Pages: 96-105
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/gt.2013.64
PMC: PMC3880610
OpenAlex: W2011980241
Cited by: 108
References: 64
Citations by year: 2026: 3 · 2025: 3 · 2024: 4 · 2023: 8 · 2022: 9 · 2021: 18 · 2020: 9 · 2019: 7 · 2018: 11 · 2017: 12 · 2016: 11 · 2015: 9 · 2014: 4
FWCI: 5.29
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Eye Institute (P30EY021721, R24EY022023, EY-021721, R01EY017549)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: adeno-associated virus vectors, retinal gene delivery, subretinal injection, intravitreal injection, canine retina, retinitis pigmentosa, AAV2, AAV8, self-complementary AAV, photoreceptor degeneration, beagle dogs
MeSH terms: Dependovirus, Animals, Capsid, Dogs, Female, Genetic Vectors, Humans, Male, Mutation, Recombinant Proteins, Retina, Transduction, Genetic, Tyrosine, Amino Acid Substitution, Viral Tropism, Injections, Intraocular

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Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

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