Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

Xin Chen, Sarah Snanoudj-Verber, Laura Pollard, Yuhui Hu, Sara S. Cathey, Ritva Tikkanen, Steven J. Gray

Research output: Contribution to journalArticlepeer-review

Abstract

Reported results demonstrated the effectiveness and safety of AAV9/AGA in Aga−/− mice, providing strong evidence that AAV9/AGA gene therapy should be considered for human translation. Moreover, the direct comparison of the efficacy of an intravenous versus intrathecal approach should greatly inform the development of similar treatments for other related disorders.

Original languageEnglish (US)
JournalMolecular Therapy
DOIs
StateAccepted/In press - 2020

Keywords

  • AAV
  • AGA
  • AGU
  • CNS
  • adeno-associated virus
  • aspartylglucosaminidase
  • aspartylglucosaminuria
  • central nervous system
  • gene therapy
  • lysosomal storage disease

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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