Title | Creator | Description | Identifier | ||
---|---|---|---|---|---|
1 | Safety/Acuity Outcomes 96-Weeks Post-Treatment with rAAV2/2-ND4; Gene Therapy for ND4 LHON: a Phase I/II Trial | Barrett Katz, Catherine Vignal, Celine Bouquet, Nitza Thomasson, Ann Galy, Serge Fitoussi, Jose Sahel | rAAV2/2-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 |