Validation of Gene Therapy for Mutant Mitochondria by Delivering Mitochondrial RNA Using a MITO-Porter

Here, we report on validating a mitochondrial gene therapy by delivering nucleic acids to mitochondria of diseased cells by a MITO-Porter, a liposome-based carrier for mitochondrial delivery. We used cells derived from a patient with a mitochondrial disease with a G625A heteroplasmic mutation in the...

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Detalles Bibliográficos
Autores principales: Kawamura, Eriko, Maruyama, Minako, Abe, Jiro, Sudo, Akira, Takeda, Atsuhito, Takada, Shingo, Yokota, Takashi, Kinugawa, Shintaro, Harashima, Hideyoshi, Yamada, Yuma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
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Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7210581/
https://www.ncbi.nlm.nih.gov/pubmed/32388194
http://dx.doi.org/10.1016/j.omtn.2020.04.004

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