Boris Kantor, PhD, Duke University School of Medicine, Durham, NC, discusses current research driving the development of next generation gene therapy techniques. Several approaches are under investigation, such as RNA interference using chemically modified small interfering RNAs (siRNAs) to repress APOE. There are concerns with this approach, however, as they have shown slow CSF clearance, CNS retention, and long-term silencing. The potent and sustained modulation of gene expression may be harmful in the case of APOE. Dr Kantor believes his approach using an AAV-based all-in-one CRISPR/dCas9 epigenome editing system holds promise, given its ability to be fine-tuned. In this way, there is a certain amount of flexibility regarding the amount of repression of the gene of interest. This interview took place at the AD/PD™ 2023 congress in Gothenburg, Sweden.
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