CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
Poojitha Pinjala1, Kamatham Pushpa Tryphena1, Renuka Prasad2
1Molecular and Cellular Neuroscience Lab, Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana-500037, Hyderabad, India.
Biomaterials Research
|May 16, 2023
Summary
CRISPR-Cas9 gene editing offers new hope for Parkinson's disease (PD) by correcting causative mutations. This technology is crucial for developing advanced stem cell therapies and disease models for genetic PD.
Area of Science:
- Neuroscience
- Genetics
- Stem Cell Biology
Background:
- Parkinson's disease (PD) remains incurable, driving research into novel genetic therapies.
- CRISPR-Cas9 gene editing technology has emerged as a powerful tool since its 2012 discovery.
- Advances in stem cell biology enable ex-vivo editing for personalized treatments.
Purpose of the Study:
- To review the significance of CRISPR-Cas9 in Parkinson's disease research.
- To highlight its role in developing accurate PD disease models.
- To explore therapeutic strategies using CRISPR-Cas9-edited stem cells.
Main Methods:
- Utilizing CRISPR-Cas9 for gene correction in PD models.
- Employing stem cell editing (embryonic and patient-derived) ex-vivo.
- Investigating pathophysiological mechanisms of genetic PD.
Main Results:
- CRISPR-Cas9 facilitates the creation of precise genetic PD models.
- Ex-vivo stem cell editing shows potential for personalized therapeutic approaches.
- Understanding disease mechanisms is key to refining gene-editing strategies.
Conclusions:
- CRISPR-Cas9-based stem cell therapy is vital for advancing Parkinson's disease research.
- This approach holds promise for both disease modeling and developing future genetic cures.
- Further elucidation of PD pathophysiology will enhance therapeutic applications.
Keywords:
Disease modelEmbryonic stem cellsGene editingHuman pluripotent stem cellsNeurodegenerative disorderα-synucleinMore Related Videos
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