ScAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution.

ABSTRACT:

Background

Adeno-associated virus (AAV)-mediated gene therapies are rapidly advancing to the clinic, and AAV engineering has resulted in vectors with increased ability to deliver therapeutic genes. Although the choice of vector is critical, quantitative comparison of AAVs, especially in large animals, remains challenging.

Methods

Here, we developed an efficient single-cell AAV engineering pipeline (scAAVengr) to simultaneously quantify and rank efficiency of competing AAV vectors across all cell types in the same animal.

Results

To demonstrate proof-of-concept for the scAAVengr workflow, we quantified - with cell-type resolution - the abilities of naturally occurring and newly engineered AAVs to mediate gene expression in primate retina following intravitreal injectio

SUBMITTER: Ozturk BE 

PROVIDER: S-EPMC8612735 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature