Gene therapy and genome editing for type i glycogen storage diseases

HIGHLIGHTS

• who: March and collaborators from the Genethon, France University College London have published the article: Gene therapy and genome editing for type I glycogen storage diseases, in the Journal: (JOURNAL)
• what: Since the studies described below are all directed by rAAV2/8 with the native human G6PC promoter/enhancer (GPE) expressing WT human G6PC protein sequence, the authors will abbreviate the construct name from ssAAV8-GPE-G6PC to rAAV-G6PC-WT for the rest of this review article.
• how: When this vector was infused into 2-week-old G6pc-/- mice at 1 x . . .
  

   

  

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