Manipulating mitochondrial genomes in the clinic: Playing by different rules

Trends in Cell Biology

Volumn 24, Issue 4, 2014, Pages 209-211

  Moraes, Carlos T ^a   Bacman, Sandra R ^a   Williams, Sion L ^a  

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

Author keywords

[No Author keywords available]

Indexed keywords

CELL NUCLEUS DNA; MITOCHONDRIAL DNA; MITOCHONDRIAL PROTEIN; NUCLEASE; RESTRICTION ENDONUCLEASE; TAL EFFECTOR NUCLEASE; TRANSFER RNA; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

CELL CLONE; CYTOPLASM; DISEASE SEVERITY; DISORDERS OF MITOCHONDRIAL FUNCTIONS; DNA SEQUENCE; DNA TRANSFECTION; EMBRYO; GENE DELETION; GENE MUTATION; GENE THERAPY; GENETIC MANIPULATION; HETEROPLASMY; HUMAN; KEARNS SAYRE SYNDROME; MAMMAL CELL; MELAS SYNDROME; MERRF SYNDROME; MITOCHONDRIAL GENOME; NONHUMAN; NOTE; OOCYTE; OXIDATIVE PHOSPHORYLATION; POINT MUTATION; PRIORITY JOURNAL; WILD TYPE; ZYGOTE; ANIMAL; GENETICS; MITOCHONDRION; MOLECULAR EVOLUTION; PATIENT RIGHT;

ANIMALS; DNA, MITOCHONDRIAL; EVOLUTION, MOLECULAR; GENOME, MITOCHONDRIAL; HUMANS; MITOCHONDRIA; PATIENT RIGHTS;

EID: 84897849651     PISSN: 09628924     EISSN: 18793088     Source Type: Journal    
DOI: 10.1016/j.tcb.2014.02.002     Document Type: Note

References (14)

1. Luo S.M., et al. Unique insights into maternal mitochondrial inheritance in mice. Proc. Natl. Acad. Sci. U.S.A. 2013, 110:13038-13043.
2. Schon E.A., et al. Therapeutic prospects for mitochondrial disease. Trends Mol. Med. 2010, 16:268-276.
3. King M.P., Attardi G. Human cells lacking mtDNA: repopulation with exogenous mitochondria by complementation. Science 1989, 246:500-503.
4. Gowher A., et al. Induced tRNA import into human mitochondria: implication of a host aminoacyl-tRNA-synthetase. PLoS ONE 2013, 8:e66228.
5. Wang G., et al. Correcting human mitochondrial mutations with targeted RNA import. Proc. Natl. Acad. Sci. U.S.A. 2012, 109:4840-4845.
6. Bacman S.R., et al. Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease. Gene Ther. 2012, 19:1101-1106.
7. Bacman S.R., et al. Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs. Nat. Med. 2013, 19:1111-1113.
8. Minczuk M., et al. Construction and testing of engineered zinc-finger proteins for sequence-specific modification of mtDNA. Nat. Protoc. 2010, 5:342-356.
9. Kaufmann K.B., et al. Gene therapy on the move. EMBO Mol. Med. 2013, 5:1642-1661.
10. Deichmann A., Schmidt M. Biosafety considerations using gamma-retroviral vectors in gene therapy. Curr. Gene Ther. 2013, 13:469-477.
11. Smeets H.J. Preventing the transmission of mitochondrial DNA disorders: selecting the good guys or kicking out the bad guys. Reprod. Biomed. Online 2013, 27:599-610.
12. Paull D., et al. Nuclear genome transfer in human oocytes eliminates mitochondrial DNA variants. Nature 2013, 493:632-637.
13. Tachibana M., et al. Towards germline gene therapy of inherited mitochondrial diseases. Nature 2013, 493:627-631.
14. Tachibana M., et al. Mitochondrial gene replacement in primate offspring and embryonic stem cells. Nature 2009, 461:367-372.