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Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants
[article]
2021
bioRxiv
pre-print
AbstractGene therapy is a rapidly developing field, and adeno-associated virus (AAV) is 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
doi:10.1101/2021.09.27.461256
fatcat:2ere4pmjq5g33plcd7vmxp2ktq