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CRISPR/Cas9 genome editing for neurodegenerative diseases
Jafar Nouri Nojadeh1,2, Nur Seren Bildiren Eryilmaz3, Berrin Imge Ergüder1,2
1Ankara University Faculty of Medicine, Department of Medical Biochemistry, Ankara, Turkey.
EXCLI Journal
|August 28, 2023
Summary
CRISPR-Cas gene editing offers precise genome modification for gene therapy. This review explores its application in treating neurodegenerative diseases like Alzheimer's and Parkinson's.
Area of Science:
- Biotechnology
- Genetics
- Neuroscience
Background:
- Gene therapy is a promising treatment for various diseases.
- Gene editing tools allow precise modification of the eukaryotic cell genome.
- CRISPR-Cas technology enables heritable genome changes via guide RNAs.
Purpose of the Study:
- To provide an overview of CRISPR-Cas technology.
- To summarize research on CRISPR/Cas9 for neurodegenerative disease treatment.
Main Methods:
- Review of current literature on CRISPR-Cas gene editing.
- Focus on CRISPR/Cas9 applications in neurodegenerative disease models.
Main Results:
- CRISPR/Cas9 is a powerful tool for targeting specific genes.
- Potential therapeutic applications for Alzheimer's, Parkinson's, Huntington's, ALS, and Spinocerebellar ataxia.
Conclusions:
- CRISPR/Cas9 technology holds significant promise for treating neurodegenerative diseases.
- Further research is needed to optimize in vivo and ex vivo gene editing strategies.
Keywords:
Alzheimer's diseaseAmyotrophic lateral sclerosisCRISPR/Cas9Huntington's diseaseParkinson's diseaseSpinocerebellar ataxiagene editingneurodegenerative disordersMore Related Videos
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