Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants.

ABSTRACT: Gene therapy is a rapidly developing field, and adeno-associated viruses (AAVs) are a leading viral-vector candidate for therapeutic gene delivery. Newly engineered AAVs with improved abilities are now entering the clinic. It has proven challenging, however, to predict the translational potential of gene therapies developed in animal models due to cross-species differences. Human retinal explants are the only available model of fully developed human retinal tissue and are thus important for the validation of candidate AAV vectors. In this study, we evaluated 18 wild-type and engineered AAV capsids in human retinal explants using a recently developed single-cell RNA sequencing (RNA-seq) AAV engineering pipeline (scAAVengr). Human retinal explants retained the same major cell types as fresh

SUBMITTER: Xi Z 

PROVIDER: S-EPMC9118357 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature