Application of CRISPR/Cas9 in Alzheimer’s Disease

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, neurof...

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Main Authors: Likui Lu, Xi Yu, Yongle Cai, Miao Sun, Hao Yang
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-12-01
Series:Frontiers in Neuroscience
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fnins.2021.803894/full
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author Likui Lu
Xi Yu
Yongle Cai
Miao Sun
Hao Yang
author_facet Likui Lu
Xi Yu
Yongle Cai
Miao Sun
Hao Yang
author_sort Likui Lu
collection DOAJ
description 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.
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spelling doaj.art-bafc63f7c1e545b280bed00312f021162022-12-21T16:58:15ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2021-12-011510.3389/fnins.2021.803894803894Application of CRISPR/Cas9 in Alzheimer’s DiseaseLikui LuXi YuYongle CaiMiao SunHao YangAlzheimer’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.https://www.frontiersin.org/articles/10.3389/fnins.2021.803894/fullgene therapyCRISPR/Cas9cognitive functionAlzheimer’s diseasedelivery system
spellingShingle Likui Lu
Xi Yu
Yongle Cai
Miao Sun
Hao Yang
Application of CRISPR/Cas9 in Alzheimer’s Disease
Frontiers in Neuroscience
gene therapy
CRISPR/Cas9
cognitive function
Alzheimer’s disease
delivery system
title Application of CRISPR/Cas9 in Alzheimer’s Disease
title_full Application of CRISPR/Cas9 in Alzheimer’s Disease
title_fullStr Application of CRISPR/Cas9 in Alzheimer’s Disease
title_full_unstemmed Application of CRISPR/Cas9 in Alzheimer’s Disease
title_short Application of CRISPR/Cas9 in Alzheimer’s Disease
title_sort application of crispr cas9 in alzheimer s disease
topic gene therapy
CRISPR/Cas9
cognitive function
Alzheimer’s disease
delivery system
url https://www.frontiersin.org/articles/10.3389/fnins.2021.803894/full
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