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Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene

Liu, Yuan, Eastwood, Jeremy D., Alba, Diego E., Velmurugan, Sindhu, Sun, Ning, Porciatti, Vittorio, Lee, Richard K., Hauswirth, William W., Guy, John, Yu, Hong·2022·Gene Therapy
DOI 10.1038/s41434-022-00333-6PMC PMC9233058
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  • Previous approaches have predominantly used recombinant adenoviruses to target specific organs, such as the eye, expressing allotopic ND4, ATP6, or ND1 transgenes.

    “Previous approaches have predominantly used recombinant adenoviruses to target specific organs, such as the eye, expressing allotopic ND4,23 ATP6,24 or ND150 transgenes.”

    From Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo (2024) · cites this paper as [50]

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Metadata

Authors
Liu, Y., Eastwood, J. D., Alba, D. E., Velmurugan, S., Sun, N., Porciatti, V., Lee, R. K., Hauswirth, W. W., Guy, J., & Yu, H.
ORCID
Venue
Gene Therapy
Publisher
Springer Science and Business Media LLC
Published
2022-04-06
Volume
29
Issue
6
Pages
368-378
ISSN
  • 0969-7128
  • 1476-5462
DOI
10.1038/s41434-022-00333-6
PMC
PMC9233058
Cited by
19
References
2
Language
en
Licence
Funders
  • U.S. Department of Health & Human Services | NIH | National Eye Institute (R24 EY028785, P30 EY014801, R01 EY 027414, R01 EY017141, R01 EY 017141)
  • R.K. Lee was partially supported by the Walter G. Ross Foundation, the Camiener Foundation Glaucoma Research Fund, and the Guiterrez Family Research Fund.
  • International Retinal Research Foundation (GR015009)

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