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

• Published in: Molecular therapy Vol. 28; no. 9; pp. 2073 - 2082
• Main Authors: George, Lindsey A., Ragni, Margaret V., Rasko, John E.J., Raffini, Leslie J., Samelson-Jones, Benjamin J., Ozelo, Margareth, Hazbon, Maria, Runowski, Alexa R., Wellman, Jennifer A., Wachtel, Katie, Chen, Yifeng, Anguela, Xavier M., Kuranda, Klaudia, Mingozzi, Federico, High, Katherine A.
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 02.09.2020; American Society of Gene & Cell Therapy
• Subjects: AAV; adeno-associated virus; clinical gene therapy; gene therapy; hemophilia; neutralizing antibody; Original; hemophilia; neutralizing antibody; gene therapy; adeno-associated virus; clinical gene therapy; AAV

Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durability, and the nature of the human immune response to...