The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer's Disease
Received Date: May 21, 2018 / Accepted Date: May 24, 2018 / Published Date: May 31, 2018
Despite a wealth of knowledge gained in the past three decades concerning the molecular underpinnings of Alzheimer’s disease (AD), progress towards obtaining effective, disease modifying therapies has proven to be challenging. In this manner, numerous clinical trials targeting the production, aggregation, and toxicity of betaamyloid, have failed to meet efficacy standards. This puts into question the beta-amyloid hypothesis and suggests that additional treatment strategies should be explored. The recent emergence of CRISPR/Cas9 gene editing as a relatively straightforward, inexpensive, and precise system has led to an increased interest of applying this technique in AD. CRISPR/Cas9 gene editing can be used as a direct treatment approach or to help establish better animal models that more faithfully mimic human neurodegenerative diseases. In this manner, this technique has already shown promise in other neurological disorders, such as Huntington’s disease. The purpose of this review is to examine the potential utility of CRISPR/Cas9 as a treatment option for AD by targeting specific genes including those that cause early-onset AD, as well as those that are significant risk factors for late-onset AD such as the apolipoprotein E4 (APOE4) gene.
Keywords: Alzheimer’s disease; CRISPR/Cas9; Gene editing; Treatment; Huntington’s disease; iPSC neurons
Citation: Rohn TT, Kim N, Isho NF, Mack JM (2018) The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer’s Disease. J Alzheimers Dis Parkinsonism 8: 439. Doi: 10.4172/2161-0460.1000439
Copyright: © 2018 Rohn TT, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
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