Application of CRISPR/Cas9 in Alzheimer’s Disease

Lu, Likui; Xi, Yu; Cai, Yongle; Sun, Miao; Yang, Hao

doi:10.3389/fnins.2021.803894

Cited by 20 publications

(6 citation statements)

References 139 publications

(151 reference statements)

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“…These models have proven to be extremely useful for understanding some aspects of AD pathogenesis, but their utility is limited because most of the AD mouse models do not develop frank neurodegeneration [ 45 ]. Recently, CRISPR-Cas9 technology has been used to create new AD models that show a more accurate disease phenotype, elucidate mechanisms of pathogenesis, screen pathogenic genes, and at the same time, find a therapy for this insidious disorder [ 46 ]. We, briefly, described below the most significant examples.…”

Section: Crispr-cas9 In Alzheimer’s Diseasementioning

confidence: 99%

Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders

Plano

Calabrese

Conoci

et al. 2022

IJMS

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Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and Huntington’s disease represent some of the most prevalent neurodegenerative disorders afflicting millions of people worldwide. Unfortunately, there is a lack of efficacious treatments to cure or stop the progression of these disorders. While the causes of such a lack of therapies can be attributed to various reasons, the disappointing results of recent clinical trials suggest the need for novel and innovative approaches. Since its discovery, there has been a growing excitement around the potential for CRISPR-Cas9 mediated gene editing to identify novel mechanistic insights into disease pathogenesis and to mediate accurate gene therapy. To this end, the literature is rich with experiments aimed at generating novel models of these disorders and offering proof-of-concept studies in preclinical animal models validating the great potential and versatility of this gene-editing system. In this review, we provide an overview of how the CRISPR-Cas9 systems have been used in these neurodegenerative disorders.

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Section: Crispr-cas9 In Alzheimer’s Diseasementioning

confidence: 99%

Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders

Plano

Calabrese

Conoci

et al. 2022

IJMS

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“…In contrast, the class 2 system, including types II, V, and VI, has an endonuclease module with a single-protein effector, which is more feasible for engineering [ 21 , 32 , 33 ]. Specifically, Cas9, as a single-chain endonuclease in the type II system, has been extensively studied to expand the genome toolkit [ 25 , 34 ].…”

Section: Crispr Function and Cellular Deliverymentioning

confidence: 99%

New Therapeutics for Extracellular Vesicles: Delivering CRISPR for Cancer Treatment

Yan

Liang

2022

IJMS

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Cancers are defined by genetic defects, which underlines the prospect of using gene therapy in patient care. During the past decade, CRISPR technology has rapidly evolved into a powerful gene editing tool with high fidelity and precision. However, one of the impediments slowing down the clinical translation of CRISPR-based gene therapy concerns the lack of ideal delivery vectors. Extracellular vesicles (EVs) are nano-sized membrane sacs naturally released from nearly all types of cells. Although EVs are secreted for bio-information conveyance among cells or tissues, they have been recognized as superior vectors for drug or gene delivery. Recently, emerging evidence has spotlighted EVs in CRISPR delivery towards cancer treatment. In this review, we briefly introduce the biology and function of the CRISPR system and follow this with a summary of current delivery methods for CRISPR applications. We emphasize the recent progress in EV-mediated CRISPR editing for various cancer types and target genes. The reported strategies for constructing EV-CRISPR vectors, as well as their limitations, are discussed in detail. The review aims to throw light on the clinical potential of engineered EVs and encourage the expansion of our available toolkit to defeat cancer.

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“…Other age-related, protective, and disease-promoting variables, such as vascular dysfunction, oxidative stress, proteinopathy, metal ions, neuroinflammation, mitochondrial dysfunction, and the microbiota-gut-brain axis, are also strongly linked to the progression of AD. The clinical signs of AD vary widely between individuals due to the etiology' intricacy [105].…”

Section: The Reason Using Crispr/cas9 As An Effective Therapy For Admentioning

confidence: 99%

A Review of CRISPR Cas9 for Alzheimer’s Disease: Treatment Strategies and Could target APOE e4, APP, and PSEN-1 Gene using CRISPR cas9 Prevent the Patient from Alzheimer’s Disease?

Adji

Widjaja²,

Wardani³

et al. 2022

Open Access Maced J Med Sci

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A Review of CRISPR Cas9 for Alzheimer’s Disease: Treatment Strategies and Could target APOE e4, APP, and PSEN-1 Gene using CRISPR cas9 Prevent the Patient from Alzheimer’s Disease? BACKGROUND: Alzheimer’s disease is a neurodegenerative disorder characterized by the formation of β-amyloid plaques and neurofibrillary tangles from hyperphosphorylated tau. Several studies suggest that targeting the deletion of the APOE e4, PSEN-1, and APP will reduce tau phosphorylation and Aβ protein accumulation, a crucial hypothesis for the causation of Alzheimer’s disease. APOE e4, PSEN-1, and APP with genome editing Clustered Regular interspersed Short Palindromic Repeats-CRISPR-related (CRISPR/Cas9) are thought to have therapeutic promise for Alzheimer’s disease.AIM: The purpose of this study was to determine whether targeting APOE e4, PSEN-1, and APP using CRISPR/Cas9 is an effective therapeutic and whether it has a long-term effect on Alzheimer’s disease.METHODS: The method used in this study summarized articles by examining the titles and abstracts of specific specified keywords. In this situation, the author picked the title and abstract that matched PubMed, Google Scholar, Science Direct, Cochrane, and the Frontiers in Neuroscience; this was followed by checking to see whether the paper was available in full-text. Eventually, the researcher will study the entire article to decide if it is valuable and relevant to the issue.RESULTS: CRISPR/Cas9 deletion of APOE e4, PSEN-1, and APP in induced pluripotent stem cells (iPSC’s) and g2576 mice as APP mutant models reduce tau phosphorylation and Aβ protein accumulation from neurofibrillary tangles and prevent cell death, vascular damage, and dementia. Furthermore, CRISPR/Cas9 deletion in APOE e4, PSEN-1, and APP improved neuronal cell resilience to oxidative stress and inflammation.CONCLUSION: APOE e4, PSEN-1, and APP deletion by genome editing CRISPR/Cas9 is effective to reduce tau phosphorylation and Aβ protein accumulation from neurofibrillary tangles, cell death, vascular damage, and dementia. However, further research is needed to determine the side effects and safety of its use.

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Application of CRISPR/Cas9 in Alzheimer’s Disease

Cited by 20 publications

References 139 publications

Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders

Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders

New Therapeutics for Extracellular Vesicles: Delivering CRISPR for Cancer Treatment

A Review of CRISPR Cas9 for Alzheimer’s Disease: Treatment Strategies and Could target APOE e4, APP, and PSEN-1 Gene using CRISPR cas9 Prevent the Patient from Alzheimer’s Disease?

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