Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
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
Topics & Concepts
Genetic enhancementMedicineFactor IXGene deliveryVirologyVector (molecular biology)Adeno-associated virusSerotypeVirusImmunologyCoagulationBiologyGeneInternal medicineRecombinant DNABiochemistryVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering