Validation of a mitochondrial RNA therapeutic strategy using fibroblasts from a Leigh syndrome patient with a mutation in the mitochondrial ND3 gene

We report on the validation of a mitochondrial gene therapeutic strategy using fibroblasts from a Leigh syndrome patient by the mitochondrial delivery of therapeutic mRNA. The treatment involves delivering normal ND3 protein-encoding mRNA as a therapeutic RNA to mitochondria of the fibroblasts from...

Descripción completa

Detalles Bibliográficos
Autores principales: Yamada, Yuma, Somiya, Kana, Miyauchi, Akihiko, Osaka, Hitoshi, Harashima, Hideyoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7200808/
https://www.ncbi.nlm.nih.gov/pubmed/32371897
http://dx.doi.org/10.1038/s41598-020-64322-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7200808/
https://www.ncbi.nlm.nih.gov/pubmed/32371897
http://dx.doi.org/10.1038/s41598-020-64322-8

Ejemplares similares