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

Therapies for genetic disorders caused by mutated mitochondrial DNA are an unmet need, in large part due barriers in delivering DNA to the organelle and the absence of relevant animal models. We injected into mouse eyes a mitochondrially targeted Adeno-Associated-Virus (MTS-AAV) to deliver the mutan...

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Detalles Bibliográficos
Autores principales: Liu, Yuan, Eastwood, Jeremy D., Alba, Diego E., Velmurugan, Sindhu, Sun, Ning, Porciatti, Vittorio, Lee, Richard K., Hauswirth, William W, Guy, John, Yu, Hong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9233058/
https://www.ncbi.nlm.nih.gov/pubmed/35383288
http://dx.doi.org/10.1038/s41434-022-00333-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9233058/
https://www.ncbi.nlm.nih.gov/pubmed/35383288
http://dx.doi.org/10.1038/s41434-022-00333-6

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