Article

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

2021-09-27

Abstract excerpt

Gene 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 availab...

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Literature Corpus work: 64f869ec-b833-5694-b4a3-3c41ee71f972
DOI: 10.1101/2021.09.27.461256

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