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Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations.

Christian HindererRod MillerCecilia DyerJulia JohanssonPeter BellElizabeth BuzaJames M Wilson
Published in: Annals of clinical and translational neurology (2020)
These findings support the development of AAV1-based gene therapy for frontotemporal dementia caused by progranulin deficiency.
Keyphrases
  • copy number
  • genome wide
  • gene therapy
  • genome wide identification
  • dengue virus
  • gene expression
  • escherichia coli