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Comparison of different gene-therapy methods to treat Leber hereditary optic neuropathy in a mouse model

INTRODUCTION: Therapies for Leber hereditary optic neuropathy (LHON), in common with all disorders caused by mutated mitochondrial DNA, are inadequate. We have developed two gene therapy strategies for the disease: mitochondrial-targeted and allotopic expressed and compared them in a mouse model of...

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Detalles Bibliográficos
Autores principales: Velmurugan, Sindhu, Chou, Tsung-Han, Eastwood, Jeremy D., Porciatti, Vittorio, Liu, Yuan, Hauswirth, William W., Guy, John, Yu, Hong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10083341/
https://www.ncbi.nlm.nih.gov/pubmed/37051151
http://dx.doi.org/10.3389/fnins.2023.1119724