Neurodegenerative Diseases Treatment Based on CRISPR

Authors

  • Hanwen Cui
  • Zeyu Zhong

DOI:

https://doi.org/10.54097/wttkcd37

Keywords:

Neurodegenerative disease, CRISPR, Application.

Abstract

Parkinson's disease (PD) is a neurodegenerative disease that is difficult to study for humans. The etiology of induced diseases is closely related to genetics neuroinflammation, mitochondrial and lysosomal dysfunction, and synaptic transport problems. The early characteristics of Alzheimer's disease (AD) are cognitive decline, behavioral changes, and language deficits. Subsequently, the patient developed comprehensive amnesia and decreased motor function, with death typically occurring within 9 years after diagnosis. CRISPR Cas9 direct therapy has been used to repair pathogenic gene mutations and as a research object or carrier for disease simulation experiments. Neuroscientists want to edit target genes and their transcripts through CRISPR/Cas9. CRISPR technology can indirectly intervene in mitochondrial autophagy to determine the regulation of the etiology of PD. Targeted activation of SAM to produce dopamine (DA) to improve motor behavior in rats. It can also mediate the allele disruption of the Swedish APP used to suppress the initial symptoms of AD. And by weakening the app-β-splitting while enhancing apps with neuroprotective effects can reduce A β the generation and manipulation of starch protein pathways control AD. The Guangxi Bama miniature pig cultivated through CRISPR/Cas9 is expected to become a large-scale animal model for studying human neurological diseases. CRISPR technology can become a reliable tool for humans to solve mutated gene diseases.

Downloads

Download data is not yet available.

References

Potting C, Crochemore C, Moretti F, et al. Genome-wide CRISPR screen for PARKIN regulators reveals transcriptional repression as a determinant of mitophagy. Proceedings of the National Academy of Sciences, 2018, 115 (2): E180-E189.

Narváez-Pérez L F, Paz-Bermúdez F, Avalos-Fuentes J A, et al. CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´ s disease. Gene Therapy, 2024, 31 (1): 31-44.

Zhu X X, Zhong Y Z, Ge Y W, et al. CRISPR/Cas9-mediated generation of Guangxi Bama minipigs harboring three mutations in α-synuclein causing Parkinson’s disease. Scientific Reports, 2018, 8 (1): 12420.

György B, Lööv C, Zaborowski M P, et al. CRISPR/Cas9 mediated disruption of the Swedish APP allele as a therapeutic approach for early-onset Alzheimer’s disease. Molecular Therapy-Nucleic Acids, 2018, 11: 429-440.

Sun J, Carlson-Stevermer J, Das U, et al. CRISPR/Cas9 editing of APP C-terminus attenuates β-cleavage and promotes α-cleavage. Nature communications, 2019, 10 (1): 53.

Bhattacherjee A, Jung J, Zia S, et al. The CD33 short isoform is a gain-of-function variant that enhances Aβ 1–42 phagocytosis in microglia. Molecular Neurodegeneration, 2021, 16: 1-22.

Downloads

Published

11-07-2024

How to Cite

Cui, H., & Zhong , Z. (2024). Neurodegenerative Diseases Treatment Based on CRISPR. Highlights in Science, Engineering and Technology, 102, 277-280. https://doi.org/10.54097/wttkcd37