Toward a therapy for mitochondrial disease

HIGHLIGHTS

• who: Biochemical Society Transactions ( et al. from the of Neurology, IRCCS, Milan, Italy have published the research: Toward a therapy for mitochondrial disease, in the Journal: (JOURNAL) of 19/Oct/2016
• what: The second group includes Adeno-associated viral (AAV)-mediated gene therapy approaches aimed at re-expressing the wild-type gene or other therapeutic genes (e_g endonucleases to shift the heteroplasmy) in targeted tissues. This approach can, however, be used only when a suitable restriction site is introduced by the mutation, as in the case of the NARP mutation, which creates a SmaI restriction . . .
  

   

  

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