Litcius/Paper detail

Discussing investigational AAV gene therapy with hemophilia patients: A guide

Robert F. Sidonio, Steven W. Pipe, Michael U. Callaghan, Leonard A. Valentino, Paul E. Monahan, Stacy E. Croteau

2020Blood Reviews53 citationsDOI

Topics & Concepts

Genetic enhancementMedicineClinical trialGene transferInvestigational DrugsAdeno-associated virusIntensive care medicineDosingHealth careBioinformaticsGeneInternal medicineBiologyGeneticsVector (molecular biology)Recombinant DNAEconomic growthEconomicsVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering