The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.
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.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“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]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“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]
Experimental — accuracy not yet measured
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Mitochondrial DNA mutation associated with Leber’s hereditary optic neuropathy
Wallace, D. C., Singh, G., Lott, M. T., Hodge, J. A., Schurr, T. G., Lezza, A. M. S., Elsas, L. J., & Nikoskelainen, E. K. (1988) · Science · doi:10.1126/science.3201231 · Cited by 2,440
Progress and prospects: gene therapy for mitochondrial DNA disease
Kyriakouli, D. S., Boesch, P., Taylor, R. W., & Lightowlers, R. N. (2008) · Gene Therapy · doi:10.1038/gt.2008.91 · Cited by 76
Flagged first, then by citations
Research works printed but not linked: 46
- 10.1007/BF01694557 · DOI
- 10.1146/annurev.neuro.30.051606.094302 · DOI
- 10.1006/bbrc.1995.1740 · DOI
- 10.1167/iovs.06-0789 · DOI · Paper page
- 10.1126/science.283.5407.1482 · DOI · Paper page
- 10.1016/j.gene.2005.05.001 · DOI
- 10.1038/440600a · DOI
- 10.1016/j.cell.2005.02.001 · DOI · Paper page
- 10.1111/j.1365-2796.2008.02058.x · DOI
- 10.1146/annurev.pathol.4.110807.092314 · DOI
- 10.1111/j.1750-3639.2000.tb00275.x · DOI
- 10.1016/j.nurt.2008.07.002 · DOI
- 10.1101/gad.1784909 · DOI
- 10.1111/j.1471-4159.2009.05867.x · DOI
- 10.1126/science.3201231 · DOI · Paper page
- 10.1016/S0042-6989(96)00167-8 · DOI
- 10.1093/nar/24.1.177 · DOI
- 10.1021/bi000335t · DOI
- 10.1038/338170a0 · DOI
- 10.1074/jbc.272.8.5342 · DOI
- 10.1093/nar/gkg228 · DOI
- 10.1023/B:JOBB.0000041773.20072.9e · DOI
- 10.1007/s11095-011-0516-4 · DOI
- 10.1016/j.mad.2011.05.002 · DOI
- 10.1093/nar/gkp348 · DOI
- 10.1093/hmg/ddi435 · DOI
- 10.1093/emboj/cdg128 · DOI
- 10.1016/j.mito.2006.12.003 · DOI
- 10.2174/156652309790031102 · DOI
- 10.1128/jvi.63.9.3822-3828.1989 · DOI
- 10.1128/JVI.78.12.6595-6609.2004 · DOI · Paper page
- 10.1002/ana.10354 · DOI · Paper page
- 10.1038/ng851 · DOI · Paper page
- 10.1093/hmg/11.4.431 · DOI
- 10.1038/mt.2008.171 · DOI · Paper page
- 10.1007/s10495-005-0742-5 · DOI
- 10.1167/iovs.08-3214 · DOI
- 10.1167/iovs.06-0789 · DOI · Paper page
- 10.1016/j.ajhg.2008.08.013 · DOI
- 10.1016/S1525-0016(03)00037-6 · DOI
- 10.1073/pnas.96.21.11770 · DOI
- 10.1016/j.bbrc.2010.05.070 · DOI
- 10.1038/nature08368 · DOI
- 10.1093/nar/gkr517 · DOI
- WW Hauswirth, et al., Production and purification of recombinant AAV vectors. Vertebrate Phototransduction and the Visual Cycle, ed K Palczewski (Academic, New York) Vol. 316, 743–761 (2000). · DOI
- 10.1016/j.ymeth.2008.09.023 · DOI
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
About these checks
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 ›
Are you an author, or do you see a problem with this page? Request a review
