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AD/PD 2023 | All-in-one AAV vector for epigenome editing shows efficient APOE downregulation in vivo

Boris Kantor, PhD, Duke University School of Medicine, Durham, NC, shares new developments from his research into APOE-targeted epigenome editing technology for Alzheimer’s disease (AD). The approach uses CRISPR/dCas9 fused to a transcriptional repressor targeted to the APOE promoter region, leading to efficient downregulation of APOE expression. In a key step forward, the team have developed an all-in-one AAV system, through comprehensive screening of Cas9 and repressor combinations. By developing a system that fits within a single vector, they have overcome several limitations of dual CRISPR/Cas9 gene editing systems, namely the reduced efficiency and requirement of a higher viral load. Using a mouse model with knock-in human APOE, administration of the all-in-one AAV- dCas9-repressor vector led to an almost 70% downregulation of APOE. The team wish to test their technology in larger animal models, having developed a non-human primate disease model using viral integration of the APP Swedish mutation and a tau mutation. 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.