Efficacy & Safety of a Tissue-Restricted AAV Vector Encoding Frataxin

Time: 11:30 am
day: Day Two: Track A AM


  • Ubiquitous overexpression of the frataxin protein, which is deficient in Friedreich’s ataxia (FA), can lead to toxicity, negating the potential therapeutic benefit of a gene therapy approach to FA
  • Tissue restricted expression of AAV-delivered frataxin is a viable approach to overcoming toxicities and achieve full therapeutic benefit
  • The efficacy and safety of this approach, from rodent models to NHPs, will be presented and discussed