Improved Genome Packaging Efficiency of Adeno-associated Virus Vectors Using Rep Hybrids.

ABSTRACT: Recombinant adeno-associated viruses (rAAVs) are one of the most commonly used vectors for a variety of gene therapy applications. In the last 2 decades, research focused primarily on the characterization and isolation of new cap, genes resulting in hundreds of natural and engineered AAV capsid variants, while the rep gene, the other major AAV open reading frame, has been less studied. This is due to the fact that the rep gene from AAV serotype 2 (AAV2) enables the single-stranded DNA packaging of recombinant genomes into most AAV serotype and engineered capsids. However, a major by-product of all vector productions is empty AAV capsids, lacking the encapsidated vector genome, especially for non-AAV2 vectors. Despite the packaging process being considered the rate-limi

SUBMITTER: Mietzsch M 

PROVIDER: S-EPMC8428402 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature