Optimized Mitochondrial Targeting of Proteins Encoded by Modified mRNAs Rescues Cells Harboring Mutations in mtATP6

Cell Reports

Volumn 22, Issue 11, 2018, Pages 2818-2826

  Chin, Randall Marcelo ^a   Panavas, Tadas ^b   Brown, Jeffrey M ^c   Johnson, Krista K ^a  

^a ALEXION PHARMACEUTICALS   (United States)

^b BOEHRINGER INGELHEIM PHARMACEUTICALS INC   (United States)

^c currently at Wave Life Sciences   (United States)

Author keywords

allotopic expression; ATP6; gene therapy; high content imaging; mitochondria; mitochondrial disease; modified mRNA; mRNA; rare disease; screening

Indexed keywords

MESSENGER RNA; MITOCHONDRIAL DNA; MITOCHONDRIAL PROTEIN;

3' UNTRANSLATED REGION; ARTICLE; AUTOMATION; CELL STRUCTURE; CONTROLLED STUDY; DNA SYNTHESIS; GENE; GENE MUTATION; MITOCHONDRIAL DYNAMICS; MITOCHONDRIAL TARGETING SIGNAL; MODIFIER GENE; MTATP6 GENE; POINT MUTATION; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; QUANTITATIVE ANALYSIS; SCREENING TEST; GENETIC TRANSFECTION; HUMAN; METABOLISM; MUTATION;

HUMANS; MITOCHONDRIAL PROTEINS; MUTATION; RNA, MESSENGER; TRANSFECTION;

EID: 85043599512     PISSN: None     EISSN: 22111247     Source Type: Journal    
DOI: 10.1016/j.celrep.2018.02.059     Document Type: Article

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