Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Authors: Lindsey A. George, Margaret V. Ragni, John E.J. Rasko, Leslie Raffini, Benjamin J. Samelson‐Jones, Margareth C. Ozelo, Maria Hazbon, Alexa R. Runowski, Jennifer Wellman, Katie Wachtel, Yifeng Chen, Xavier M. Anguela, Klaudia Kuranda, Federico Mingozzi, Katherine A. High - Molecular Therapy 2020 cited by 209