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- FXN gene therapy / AAVANTIBio
Prevention of Cardiac Disease Features Observed in a Conditional Knockout Mouse Model of Friedreich’s Ataxia Treated with a Novel AAV FXN Gene Therapy (AVB-202) (Poster Board Number: W-46; Hall D) - Apr 20, 2022 - Abstract #ASGCT2022ASGCT_616;
Additional molecular analysis is ongoing to characterize recovery of frataxin protein levels and of mitochondrial function in heart tissue. Our findings demonstrate strong positive effects on survival and cardiac function in this FA model and support continued development of AVB-202.