Preclinical Research on CRISPR-Based Gene Editing for Alzheimer Disease Treatment: Brent D. Aulston, PhD

WATCH TIME: 5 minutes

"We target a sequence that is relevant for both familial AD, and also applicable to sporadic AD. In terms of precision medicine, we're not targeting some subset of patients with AD, we're designing this in a way that can be applicable to any patient with the disease. That's why it was exciting to us, and hoping this would be relevant for all patients with AS.”

Current research on the pathology of Alzheimer disease (AD) has been studied for decades, yet there are no effective disease-modifying therapies , and existing treatments have led to irreversible and progressive cognitive decline in patients.¹ CRISPR-Cas9 technology, precise and effective gene editing, has allowed the development of empirical AD models, therapeutic strokes, and diagnostic approaches to better understand the nervous system, from in vitro to in vivo models.^2,3

Brent D. Aulston, PhD, postdoctoral fellow at the University of California San Diego, presented findings from the Subhojit Roy lab on the safety and efficacy of CRISPR-based gene therapy in mice models at the 2023 Alzheimer’s Association International Conference, July 16-20, in Amsterdam, the Netherlands.4 Prior to the meeting, Aulston sat down in an interview with NeurologyLive® to discuss more about the results from the lab using CRISPR for AD.

Aulston talked about how the CRISPR gene editing technique affects the accumulation of amyloid beta plaques in the brains of AD in mice. He also spoke about the next steps in the development of the CRISPR tool for targeting the specific human gene sequence relevant to AD. In addition, he explained why the ability of the CRISPR therapeutic to target a sequence applicable to both familial and sporadic AD considered significant.

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REFERENCES
1. Lu L, Yu X, Cai Y, Sun M, Yang H. Application of CRISPR/Cas9 in Alzheimer's Disease. Front Neurosci. 2021;15:803894. Published 2021 Dec 21. doi:10.3389/fnins.2021.803894
2. Guan L, Han Y, Yang C, et al. CRISPR-Cas9-Mediated Gene Therapy in Neurological Disorders. Mol Neurobiol. 2022;59(2):968-982. doi:10.1007/s12035-021-02638-w
3. Bhardwaj S, Kesari KK, Rachamalla M, et al. CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics. J Adv Res. 2022;40:207-221. doi:10.1016/j.jare.2021.07.001
4. Aulston BD, Branes K, Checka N, Parra-Rivas LA, Roy S. In vivo safety and efficacy of a CRISPR-based gene therapy for Alzheimer's disease. Presented at: 2022 Alzheimer’s Association International Conference; July 16 to July 20; Amsterdam, the Netherlands.