Ensuring the safety of epigenome therapies in the treatment of Alzheimer’s disease

Ornit Chiba-Falek, PhD, Duke University School of Medicine, Durham, NC, and her team have recently developed an epigenome therapy to reduce the expression of APOE, specifically the APOEe4 allele, to treat late onset Alzheimer’s disease (LOAD). The therapy, which uses CRISPR/dCas9 gene editing technology, has elicited promising reductions in APOE-mRNA levels in hiPSC-derived neuronal models and in mice hippocampi. When developing a gene editing technology such as this, there are certain safety considerations that should be taken into account, particularly concerning the specificity and delivery method of the gene therapy. Dr Chiba-Falek presents some of these considerations within the context of this new epigenome therapy. Firstly, the therapy will be delivered using adeno-associated viruses (AAVs), which are considered to be one of the safer viral gene delivery methods in terms of genotoxicity and immunotoxicity. Secondly, the therapy will not completely eliminate the expression of APOE, but rather reduce its expression. Lastly, it will be not only allele-specific but cell-specific, which reduces the chance of off-target effects. This interview took place at the Clinical Trials on Alzheimer’s Disease Congress 2022 in San Francisco.

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