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CRISPR/Cas9 genome editing for neurodegenerative diseases

Jafar Nouri Nojadeh, Nur Seren Bildiren Eryilmaz, Berrin İmge Ergüder

2023PubMed46 citationsDOIOpen Access PDF

Abstract

approaches. Gene editing tools, such as zinc finger nucleases, transcription activator-like effector nucleases, and CRISPR-Cas-associated nucleases, can be employed for gene therapy purposes. Among these tools, CRISPR-Cas-based gene editing stands out because of its ability to introduce heritable genome changes by designing short guide RNAs. This review aims to provide an overview of CRISPR-Cas technology and summarizes the latest research on the application of CRISPR/Cas9 genome editing technology for the treatment of the most prevalent neurodegenerative diseases including Alzheimer's disease, Parkinson's disease, Huntington's disease, Amyotrophic lateral sclerosis, and Spinocerebellar ataxia.

Topics & Concepts

CRISPRGenome editingZinc finger nucleaseCas9Genetic enhancementTranscription activator-like effector nucleaseBiologyGenomeComputational biologyGeneGuide RNASpinocerebellar ataxiaBioinformaticsGeneticsCRISPR and Genetic EngineeringGenetics, Aging, and Longevity in Model OrganismsPluripotent Stem Cells Research
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