Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?
Takashi Kishimoto, R. Jude Samulski
Abstract
KEYWORDS: Gene therapyadeno-associated virusAAV toxicityhepatotoxicitythrombotic microangiopathyhepatocellular carcinomadorsal root ganglia toxicityAAV capsid engineeringimmune tolerance
Topics & Concepts
ToxicityMedicinePharmacologyBiologyInternal medicineVirus-based gene therapy researchCAR-T cell therapy researchRNA Interference and Gene Delivery