A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism.

ABSTRACT: Adeno-associated virus (AAV) capsid modification enables the generation of recombinant vectors with tailored properties and tropism. Most approaches to date depend on random screening, enrichment, and serendipity. The approach explored here, called BRAVE (barcoded rational AAV vector evolution), enables efficient selection of engineered capsid structures on a large scale using only a single screening round in vivo. The approach stands in contrast to previous methods that require multiple generations of enrichment. With the BRAVE approach, each virus particle displays a peptide, derived from a protein, of known function on the AAV capsid surface, and a unique molecular barcode in the packaged genome. The sequencing of RNA-expressed barcodes from a single-generation in vivo screen allows the

SUBMITTER: Davidsson M 

PROVIDER: S-EPMC6936499 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature