TY - JOUR
T1 - Validation of Gene Therapy for Mutant Mitochondria by Delivering Mitochondrial RNA Using a MITO-Porter
AU - Kawamura, Eriko
AU - Maruyama, Minako
AU - Abe, Jiro
AU - Sudo, Akira
AU - Takeda, Atsuhito
AU - Takada, Shingo
AU - Yokota, Takashi
AU - Kinugawa, Shintaro
AU - Harashima, Hideyoshi
AU - Yamada, Yuma
N1 - Funding Information:
We thank Dr. Milton Feather for his helpful advice in writing the manuscript. This work was supported by Grants-in-Aid for Scientific Research (B) ( 26282131 and 17H02094 to Y.Y.) from the Ministry of Education, Culture, Sports, Science and Technology and Japanese Government (MEXT) .
Funding Information:
G625A fibroblasts were obtained from a mitochondrial disease patient at the Sapporo City General Hospital. The G625A fibroblasts carry a heteroplasmic mutation in the tRNA for phenylalanine in the mitochondrial DNA, leading to a decreased complex III activity. The phenotype includes epilepsy, hearing loss, and elevated lactate levels. 5 The normal human skin fibroblasts, NB1RGB (RCB0222), were provided by the Riken BioResource Research Center (BRC) through the National Bio-Resource Project of the Ministry of Education, Culture, Sports, Science and Technology (MEXT)/Japan Agency for Medical Research and Development (AMED), Japan. The G625A fibroblasts and NB1RGB were maintained in DMEM with high glucose and α-MEM, respectively. The mediums contained 10% FBS, supplemented with penicillin and streptomycin. Cells were grown in 10 cm dishes at 37°C under 5% CO 2 until reaching approximately 80% confluence. Cell passage was performed every 2–4 days.
Publisher Copyright:
© 2020 The Authors
PY - 2020/6/5
Y1 - 2020/6/5
N2 - A mitochondrial gene therapy was validated by mitochondrial delivery of a therapeutic RNA in diseased cells with a mutation in the mitochondrial RNA using a MITO-Porter, a nanocarrier for mitochondrial delivery. Mitochondrial transfection of the therapeutic RNA decreased the mutation rate and improved mitochondrial function of the disease cells.
AB - A mitochondrial gene therapy was validated by mitochondrial delivery of a therapeutic RNA in diseased cells with a mutation in the mitochondrial RNA using a MITO-Porter, a nanocarrier for mitochondrial delivery. Mitochondrial transfection of the therapeutic RNA decreased the mutation rate and improved mitochondrial function of the disease cells.
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U2 - 10.1016/j.omtn.2020.04.004
DO - 10.1016/j.omtn.2020.04.004
M3 - Article
AN - SCOPUS:85084242479
SN - 2162-2531
VL - 20
SP - 687
EP - 698
JO - Molecular Therapy - Nucleic Acids
JF - Molecular Therapy - Nucleic Acids
ER -