Article

scAAVengr: Single-cell transcriptome-based quantification of engineered AAVs in non-human primate retina

2020-10-02

Abstract excerpt

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. Here, we developed an efficient single-cell AAV engineering pipeline (scAAVengr) to quantify efficiency...

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Literature Corpus work: f32ed9fa-a6b2-5dc1-adb2-061443d39cf8
DOI: 10.1101/2020.10.01.323196

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