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 - 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.
UR - http://www.scopus.com/inward/record.url?scp=85084242479&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85084242479&partnerID=8YFLogxK
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 -