Abstract
Alzheimer’s disease (AD) is a progressive and irreversible neurodegenerative disorder clinically characterized by cognitive impairment, abnormal behavior, and social deficits, which is intimately linked with excessive β-amyloid (Aβ) protein deposition along with many other misfolded proteins, neurofibrillary tangles formed by hyperphosphorylated tau protein aggregates, and mitochondrial damage in neurons, leading to neuron loss. Currently, research on the pathological mechanism of AD has been elucidated for decades, still no effective treatment for this complex disease was developed, and the existing therapeutic strategies are extremely erratic, thereby leading to irreversible and progressive cognitive decline in AD patients. Due to gradually mental dyscapacitating of AD patients, AD not only brings serious physical and psychological suffering to patients themselves, but also imposes huge economic burdens on family and society. Accordingly, it is very imperative to recapitulate the progress of gene editing-based precision medicine in the emerging fields. In this review, we will mainly focus on the application of CRISPR/Cas9 technique in the fields of AD research and gene therapy, and summarize the application of CRISPR/Cas9 in the aspects of AD model construction, screening of pathogenic genes, and target therapy. Finally, the development of delivery systems, which is a major challenge that hinders the clinical application of CRISPR/Cas9 technology will also be discussed.
Highlights
Alzheimer’s disease (AD) is a progressive and irreversible neurodegenerative disorder that is characterized by cognitive dysfunction, abnormal behavior, social deficits, and an eventual inability to perform daily tasks
The CRISPR/Cas9 system has been successfully applied to modify the genomes of different animals including mouse (Komor et al, 2016; Serneels et al, 2020; Takalo et al, 2020), rat (Serneels et al, 2020), monkey (Niu et al, 2014; Chen et al, 2015), cell lines including HT22 (Wang et al, 2019), N2a (Sun et al, 2017), stem cells (Paquet et al, 2016)
There are already many publications on AD-related research mediated by CRISPR/Cas9, which mainly involves the use of this technology to construct AD model, screen pathogenic genes and treat AD via specific target genes
Summary
Alzheimer’s disease (AD) is a progressive and irreversible neurodegenerative disorder that is characterized by cognitive dysfunction, abnormal behavior, social deficits, and an eventual inability to perform daily tasks. CRISPR/Cas9 technology is increasingly mature and it has the potential to become an effective tool for gene editing in clinical therapy. Due to the short experimental period and relatively low consumption of CRISPR/Cas9 technology, CRISPR/Cas9 is currently widely used in the AD field including construction of AD model, screening pathogenic genes, and target therapy.
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