Dual-function aav gene therapy reverses late-stage canavan disease pathology in mice

HIGHLIGHTS

• who: ufeffDominikufeff ufeffFru00f6hlichufeff and collaborators from the University College London, United Kingdom have published the paper: Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice, in the Journal: (JOURNAL)
• what: The authors investigated potential added benefits of a dual-function vector approach aiming at combining ASPA gene addition and, at the same time, lowering Nat8l expression. The authors aimed at corroborating in_vivo the results obtained with dual-function shRNA and miRNA vectors in primary neurons. The authors attempted to push the time point of intervention to the maximum. Taken together, this . . .
  

   

  

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