CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease <i>In Vivo</i>

Yoon, Hyun Soo; Ye, Sunghyeok; Lim, Sunhwa; Jo, Ara; Lee, Hawon; Hong, Felix; Lee, Seung Eun; Oh, Soo‐Jin; Kim, Na-Rae; Kim, Kyoungmi; Kim, Bumjoon J.; Kim, Hyun‐Jin; Lee, C Justin; Nam, Min‐Ho; Hur, Junseok W.; Jeon, Sang Ryong

doi:10.1089/crispr.2021.0025

Cited by 31 publications

(18 citation statements)

References 52 publications

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“…Overexpression of α-synuclein, motor symptoms, dopaminergic neurodegeneration, and reactive microgliosis was reduced dramatically when the A53T-SNCA gene was deleted. The findings support the use of the CRISPR-Cas9 technique to minimize A53T-SNCA-specific PD [110]. Furthermore, Y Chen et al used the CRISPR-Cas9n strategy to establish SNCA−/− and SNCA+/− cell lines by deleting the endogenous…”

Section: Gene Therapy and Pdsupporting

confidence: 57%

CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease

et al. 2022

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Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques hold great promise for research and therapy of NDs, such as PD, for which there are currently no effective disease-modifying treatments. As a result, gene therapy may offer new treatment options, transforming our ability to treat this disease. We present a detailed overview of novel gene-editing delivery vehicles, which is essential for their successful implementation in both cutting-edge research and prospective therapeutics. Moreover, we review the most recent advancements in CRISPR-based applications and gene therapies for a better understanding of treating PD. We explore the benefits and drawbacks of using them for a range of gene-editing applications in the brain, emphasizing some fascinating possibilities.

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Section: Gene Therapy and Pdsupporting

confidence: 57%

CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease

et al. 2022

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“…The results of those studies showed that the adeno-associated virus carrying SaCas9-KKH with a sgRNA effectively targeting A53T-SNCA and significantly decreased the expression of A53T-SNCA in the cell line. Moreover, A53T-SNCA deletion significantly released the αsynuclein up expression, dopaminergic neurodegeneration, reactive microgliosis, and parkinsonian motor indicators (Yoon et al, 2022). According to this outcome, we supped that the CRISPR/Cas9 scheme can provide a potentiality inhibition policy for A53T-SNCA-specific in PD.…”

Section: Parkinson's Disease (Pd)mentioning

confidence: 72%

Applying the CRISPR/Cas9 for Treating Human and Animal Diseases – Comprehensive Review

Abdelnour

Salaka

Shakoori

et al. 2023

Annals of Animal Science

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Recently, genome editing tools have been extensively used in many biomedical sciences. The gene editing system is applied to modify the DNA sequences in the cellular system to comprehend their physiological response. A developing genome editing technology like clustered regularly short palindromic repeats (CRISPR) is widely expended in medical sciences. CRISPR and CRISPR-associated protein 9 (CRISPR/Cas9) system is being exploited to edit any DNA mutations related to inherited ailments to investigate in animals (in vivo) and cell lines (in vitro). Remarkably, CRISPR/Cas9 could be employed to examine treatments of many human genetic diseases such as Cystic fibrosis, Tyrosinemia, Phenylketonuria, Muscular dystrophy, Parkinson’s disease, Retinoschisis, Hemophilia, β-Thalassemia and Atherosclerosis. Moreover, CRISPR/Cas9 was used for disease resistance such as Tuberculosis, Johne’s diseases, chronic enteritis, and Brucellosis in animals. Finally, this review discusses existing progress in treating hereditary diseases using CRISPR/Cas9 technology and the high points accompanying obstacles.

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“…Notably, this was associated with about 50% drop in neurotoxic inclusions, about 15% increase in lifespan and a significant improvement in certain motor functions. As for autosomal dominant PD, deletions of synuclein alpha (SNCA) (encodes for α-synuclein protein) and leucine-rich repeat kinase 2 (LRRK2) mutant (responsible for late onset-PD) were the target strategies of recent studies (55,58). CRISPR-Cas9 engineered isogenic iPSCs to produce surviving dopaminergic neurons with reduced LRRK2 kinase activation and phospho-αsynuclein expression (55) What should be done in the next 10 years?…”

Section: Neurodegenerative Disordersmentioning

confidence: 99%

What did CRISPR-Cas9 accomplish in its first 10 years?

Khlidj

2023

Biochem. med. (Online)

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It’s been 10 years now from the debut of clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR/Cas9) era in which gene engineering has never been so accessible, precise and efficient. This technology, like a refined surgical procedure, has offered the ability of removing different types of disease causing mutations and restoring key proteins activity with ease of outperforming the previous resembling methods: zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). Additionally, CRISPR-Cas9 systems can systematically introduce genetic sequences to the specific sites in the human genome allowing to stimulate desired functions such as anti-tumoral and anti-infectious faculties. The present brief review provides an updated resume of CRISPR-Cas9’s top achievements from its first appearance to the current date focusing on the breakthrough research including in vitro, in vivo and human studies. This enables the evaluation of the previous phase ‘the proof-of-concept phase’ and marks the beginning of the next phase which will probably bring a spate of clinical trials.

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CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease In Vivo

Cited by 31 publications

References 52 publications

CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease

CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease

Applying the CRISPR/Cas9 for Treating Human and Animal Diseases – Comprehensive Review

What did CRISPR-Cas9 accomplish in its first 10 years?

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