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

• Main Authors: Eriko Kawamura (Author), Minako Maruyama (Author), Jiro Abe (Author), Akira Sudo (Author), Atsuhito Takeda (Author), Shingo Takada (Author), Takashi Yokota (Author), Shintaro Kinugawa (Author), Hideyoshi Harashima (Author), Yuma Yamada (Author)
• Format: Book
• Published: Elsevier, 2020-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...