The associate research professor of neurobiology at Duke University discussed an innovative epigenome editing approach that shows promising prospects for patient improvement, disease prevention, and potential use in Alzheimer disease prophylactic work. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"Our epigenome editing approach ensures no changes to the DNA, avoiding the risks associated with conventional gene editing techniques. This novel approach holds significant promise in improving patient outcomes, preventing diseases, and possibly offering Alzheimer's prophylactic treatment in the future."
In research, some genes have been reported to heighten the likelihood of developing Alzheimer disease (AD). Among these genetic risk factors, the most notable one is known as apolipoprotein (APOE)e4. Although inheriting this gene does not necessarily guarantee the onset of the disease, possessing a copy of this allele raises the risk of developing AD by 2 to 3 times more. Also, if an individual has 2 copies of APOE-e4 genes, the risk increases even further, approximately 8 to 12 times.1
Recently, lead author Boris Kantor, PhD, associate research professor of neurobiology at Duke University, and colleagues, presented findings on an epigenome therapy platform based on the CRISPR/dCas9-editing strategy at the 2023 Alzheimer’s Association International Conference, July 16-20, in Amsterdam, the Netherlands.2 In the study, epigenome editing reduced the levels of APOE-e4 in human induced pluripotent stem cells obtained in miniature brains from a patient with AD and humanized mouse models. Notably, this was performed without modifying the levels of other APOE variants that are thought to be neutral or protective.1
Kantor sat down in an interview with NeurologyLive® to discuss the benefits of researching this innovative approach. He talked about how the epigenome editing approach is different from traditional gene editing methods in terms of DNA alterations and enzyme activity. He also about the advantages the activated Cas9 offers in terms of DNA damage, off-target effects, and early intervention. In addition, Kantor shared his thoughts on how epigenome editing might potentially pave the way for disease prevention and AD prophylactic treatment using genetic testing and biomarkers.