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

Bilge E Öztürk1, Molly E Johnson1, Michael Kleyman2

  • 1Department of Ophthalmology, University of Pittsburgh, Pittsburgh, United States.

Elife
|October 19, 2021
PubMed
Summary

A new single-cell AAV engineering pipeline (scAAVengr) efficiently quantifies and ranks adeno-associated virus (AAV) vector performance in vivo. This tool accelerates the development of AAV gene therapies by identifying superior vectors for therapeutic applications.

Keywords:
adeno-associated viruscallithrix jacchusmacaca fascicularismedicineneuroscienceretinal degenerationrhesus macaquescRNA-Seq

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