Improved Genome Packaging Efficiency of Adeno-associated Virus Vectors Using Rep Hybrids
Mario Mietzsch, Courtnee Eddington, Ariana Jose, Jane Hsi, Paul R. Chipman, Tom Henley, Modassir Choudhry, Robert McKenna, Mavis Agbandje‐McKenna
Abstract
gene therapies if not removed by additional purification steps. Thus, there is a need to increase the genome packaging efficiency and reduce the number of empty capsids from AAV biologics. The novel Rep hybrids from different AAV serotypes described in this study are capable of reducing the percentage of empty capsids in all tested AAV serotypes and improve overall yields of genome-containing AAV capsids at the same time. They can likely be integrated easily into existing AAV manufacturing protocols to optimize the production of the generated AAV gene therapy products.
Topics & Concepts
CapsidBiologyAdeno-associated virusGenomeVirologyGeneGeneticsVector (molecular biology)VirusRecombinant DNAComputational biologyVirus-based gene therapy researchCRISPR and Genetic EngineeringCAR-T cell therapy research