Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
Glenn F. Pierce, Sylvia Fong, Brian Long, Radosław Kaczmarek
Topics & Concepts
Adeno-associated virusGenetic enhancementVirusMedicineFactor IXDiseaseLiver diseaseBiologyVectors in gene therapyGeneImmunologyBioinformaticsViral vectorVector (molecular biology)GeneticsInternal medicineRecombinant DNAVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering