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AD/PD 2023 | Benefits of an all-in-one AAV CRISPR/Cas9 system for genome/epigenome editing

Boris Kantor, PhD, Duke University School of Medicine, Durham, NC, comments on the limitations resulting from the restricted cargo capacity of adeno-associated virus (AAV) vectors and how they are working to overcome this with all-in-one CRISPR/Cas epigenome editing systems. AAV vectors are very commonly used given their safety, but they have a small packaging capacity, making them difficult to use for bulky cargo. Previous studies have used a two-vector CRISPR system for epigenome editing. However, this approach is limited by its need for a higher viral load and its limited efficiency. Dr Kantor and his team have conducted comprehensive screening to identify an effective Cas9-repressor combination capable of targeting APOE but small enough to fit in a single viral particle. Their all-in-one AAV-dCas9-repressor system led to an almost 70% downregulation of APOE in animal model studies. Additionally, it has shown that the epigenome editing effects remained in dividing cells, even when no AAV genomes remained. This interview took place at the AD/PD™ 2023 congress in Gothenburg, Sweden.

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The discussed work is subject to US Provisional Patent Appl. DU6741PCT.