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Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model
Abstract
To introduce DNA into mitochondria efficiently, we fused adenoassociated virus capsid VP2 with a mitochondrial targeting sequence to carry the mitochondrial gene encoding the human NADH ubiquinone oxidoreductase subunit 4 ( ND4 ). Expression of WT ND4 in cells with the G11778A mutation in ND4 led to restoration of defective ATP synthesis. Furthermore, with injection into the rodent eye, human ND4 DNA levels in mitochondria reached 80% of its mouse homolog. The construct expressed in most inner retinal neurons, and it also suppressed visual loss and optic atrophy induced by a mutant ND4 homolog. The adenoassociated virus cassette accommodates genes of up to ∼5 kb in length, thus providing a platform for introduction of almost any mitochondrial gene and perhaps even allowing insertion of DNA encompassing large deletions of mtDNA, some associated with aging, into the organelle of adults.
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“Despite these difficulties, some animal studies targeting the ATP6 and ND4 genes using AE have shown promising data [128,165,166,167].”
From The Mitochondrial Genome in Aging and Disease and the Future of Mitochondrial Therapeutics (2022) · cites this paper as [166]
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“Similarly, mutant versions of the ND4 and ND6 genes have been expressed using mitochondria-targeted AAV, to induce LHON phenotypes in mouse models [151,165,166,168].”
From The Mitochondrial Genome in Aging and Disease and the Future of Mitochondrial Therapeutics (2022) · cites this paper as [166]
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Cites
Mitochondrial DNA mutation associated with Leber’s hereditary optic neuropathy
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Progress and prospects: gene therapy for mitochondrial DNA disease
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Cited by
Sorted by citations
A comprehensive review of retinal gene therapy
Boye, S. E., Boye, S. L., Lewin, A. S., & Hauswirth, W. W. (2013) · Molecular Therapy · doi:10.1038/mt.2012.280 · Cited by 262
Self-citationCapsid modifications for targeting and improving the efficacy of AAV vectors
Büning, H., & Srivastava, A. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.01.008 · Cited by 260
Gene Therapy for Leber Hereditary Optic Neuropathy
Feuer, W. J., Schiffman, J. C., Davis, J. L., Porciatti, V., Gonzalez, P., Koilkonda, R. D., Yuan, H., Lalwani, A., Lam, B. L., & Guy, J. (2016) · Ophthalmology · doi:10.1016/j.ophtha.2015.10.025 · Cited by 230
Self-citationBasic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balakrishnan, B., & Jayandharan, G. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140302193709 · Cited by 225
Leber hereditary optic neuropathy: current perspectives.
McClelland, C., Meyerson, C., & Van Stavern, G. (2015) · Clinical Ophthalmology · doi:10.2147/opth.s62021 · Cited by 196
Metadata
- Authors
- Yu, H., Koilkonda, R. D., Chou, T.-H., Porciatti, V., Ozdemir, S. S., Chiodo, V., Boye, S. L., Boye, S. E., Hauswirth, W. W., Lewin, A. S., & Guy, J.
- Affiliations
- Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami, Miami, FL 33136; and
- Departments of bOphthalmology and
- Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, FL 32610
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2012-04-20
- Volume
- 109
- Issue
- 20
- ISSN
- 0027-8424
- 1091-6490
- Cited by
- 154
- References
- 2
- Language
- en
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