MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases

Molecular Therapy

Volumn 23, Issue 10, 2015, Pages 1592-1599

  Hashimoto, Masami ^a   Bacman, Sandra R ^a   Peralta, Susana ^a   Falk, Marni J ^b   Chomyn, Anne ^c   Chan, David C ^c   Williams, Sion L ^a,d   Moraes, Carlos T ^a  

^a UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c California Institute of Technology   (United States)

^d UNIVERSITY OF MIAMI   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MITOCHONDRIAL DNA; DEOXYRIBONUCLEASE; MITOCHONDRIAL PROTEIN; ND5 PROTEIN, HUMAN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE DEHYDROGENASE (UBIQUINONE); TRANSCRIPTION FACTOR;

AMINO TERMINAL SEQUENCE; ARTICLE; CONTROLLED STUDY; DISORDERS OF MITOCHONDRIAL FUNCTIONS; ENZYME ACTIVITY; GENE SEQUENCE; GENETIC ASSOCIATION; HETEROPLASMY; HUMAN; HUMAN CELL; LEIGH DISEASE; MELAS SYNDROME; MERRF SYNDROME; MITOCHONDRIAL TARGETED TALEN; MOLECULAR BIOLOGY; MOLECULAR RECOGNITION; OXIDATIVE PHOSPHORYLATION; POINT MUTATION; UPREGULATION; ANIMAL; CELL LINE; GENE DOSAGE; GENE EXPRESSION; GENE ORDER; GENE THERAPY; GENE VECTOR; GENETICS; HYDROLYSIS; METABOLISM; MITOCHONDRIAL DISEASES; MITOCHONDRION; MUTATION; PROTEIN TRANSPORT;

ANIMALS; CELL LINE; DEOXYRIBONUCLEASES; DNA, MITOCHONDRIAL; ELECTRON TRANSPORT COMPLEX I; GENE DOSAGE; GENE EXPRESSION; GENE ORDER; GENETIC THERAPY; GENETIC VECTORS; HUMANS; HYDROLYSIS; MITOCHONDRIA; MITOCHONDRIAL DISEASES; MITOCHONDRIAL PROTEINS; MUTATION; OXIDATIVE PHOSPHORYLATION; POINT MUTATION; PROTEIN TRANSPORT; TRANSCRIPTION FACTORS;

EID: 84943584938     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.126     Document Type: Article

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