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Article

Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor.

Nucleic acid therapeutics - 1 Jun 2023

Wimmer Tobias, Sawinski Hannah, Urban Anne M, Motlik Jan, Stieger Knut

Abstract excerpt

Stargardt disease (STGD) leads to blindness in children and young adults. So far, no curative therapy is available and gene augmentation therapies have not yet advanced to the clinics, in part, due to the limited packaging capacity of adeno-associated viruses used to transfer genes into photoreceptor cells. Prime editing offers a new perspective to treat mutations on the genomic level. A nicking variant of Cas9...

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Identifiers

PMID: 36857739
DOI: 10.1089/nat.2022.0037
PMCID: PMC10278032

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