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TitleCreatorDescriptionIdentifier
1 Safety/Acuity Outcomes 96-Weeks Post-Treatment with rAAV2/2-ND4; Gene Therapy for ND4 LHON: a Phase I/II TrialBarrett Katz, Catherine Vignal, Celine Bouquet, Nitza Thomasson, Ann Galy, Serge Fitoussi, Jose SahelrAAV2/2-ND4 is an investigational gene therapy enabling allotopic transgene expression. We report safety and visual acuity outcomes 96 weeks post-treatment in a Phase I/II open-label dose escalation trial.20180305_nanos_sciplatform1_08
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