Novel aav-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia

HIGHLIGHTS

  • who: Shengwen Zhang and colleagues from the Editor: Chen Ling, Fudan University, CHINA have published the Article: Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia, in the Journal: PLOS ONE of September/20,/2022
  • what: The aim of this experiment was to evaluate the effect of low protein diet on the survival of the MMA mice without any other treatment. Because the study was performed in PXB mice with wild-type human hepatocytes, there was no selective advantage to drive the expansion of the edited hepatocytes . . .

     

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