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Adeno-associated viral vector serotype 1 (AAV1) gene therapy for FTD-GRN: A phase 1b dose-escalation study to assess safety, tolerability, and pharmacodynamic effects of PBFT02

Voss T, Grossman M, Irwin DJ, Schulz PE, de Souza LC, Triglia P, Quadrini KJ, Nagilla P, Forman MS

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