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A Broad Application of CRISPR Cas9 in Infectious, Inflammatory and Neurodegenerative Diseases
1Division of Research and Development, Jesse Brown Veterans Affairs Medical Center, 820 S. Damen Ave, Chicago, IL 60612, USA. Kalipada_Pahan@rush.edu.
Abstract:
Being the most important immune-responsive cell type of the CNS, microglia always glorify the so-called crossroad of Neurology, Immunology and Pharmacology. As microglial activation is a hallmark of different neurodegenerative disorders including Alzheimer's disease (AD), Parkinson's disease (PD), HIV-associated neurocognitive disorders (HAND), Amyotrophic lateral sclerosis (ALS), etc., selective targeting of microglial cell signaling may be a valid option to control these neurodegenerative disorders with lesser side effects. This is particularly important as no effective therapies are available against these diseases and available neuroimmune modulators are known to target multiple cell types in a non-cell-specific manner. How we can achieve such specificity? A newly-developed cutting-edge molecular biology tool is rocking biomedical research in recent years so much so that it has already come under major lawsuits between the University of California Berkeley and the MIT-Harvard Broad Institute regarding its ownership rights, probably halting the Nobel committee to announce the most coveted prize to its owners. It is none other than Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR). In nutshell, the Cas9 enzyme has been paired with the bacterial immune system, CRISPR, to ultimately turn CRISPR/Cas9 as an effective genome editor. Therefore, this special issue has been devoted to highlight some of the recent discoveries on CRISPR/Cas9 in neurodegenerative disorders and explain these discoveries in the light of neuroimmune pharmacology.
Insights
Microglia, key immune cells in the central nervous system, are central to neurodegenerative diseases. CRISPR/Cas9 technology offers a novel approach for targeted microglial signaling modulation to treat these conditions.
Area of Science:
- Neuroimmunology
- Neuropharmacology
- Molecular Biology
Background:
- Microglia are the primary immune cells in the central nervous system (CNS).
- Microglial activation is a common feature in neurodegenerative diseases like Alzheimer's disease (AD), Parkinson's disease (PD), and HIV-associated neurocognitive disorders (HAND).
- Current treatments for neurodegenerative diseases are limited, and existing neuroimmune modulators lack cell specificity.
Purpose of the Study:
- To explore the potential of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 technology for targeted microglial signaling.
- To highlight recent advancements in CRISPR/Cas9 applications for neurodegenerative disorders.
- To discuss these advancements within the context of neuroimmune pharmacology.
Main Methods:
- Review of recent scientific literature on CRISPR/Cas9 applications in neurodegenerative disease research.
- Analysis of CRISPR/Cas9's mechanism as a genome editing tool.
- Focus on neuroimmune pharmacology and cell-specific targeting strategies.
Main Results:
- CRISPR/Cas9 technology has emerged as a powerful tool for precise genome editing.
- This technology holds promise for selectively targeting microglial cell signaling pathways.
- Potential for developing novel therapeutic strategies for neurodegenerative disorders with improved specificity.
Conclusions:
- Targeting microglial signaling with CRISPR/Cas9 offers a promising avenue for treating neurodegenerative diseases.
- The specificity of CRISPR/Cas9 can overcome the limitations of current non-specific neuroimmune modulators.
- Further research into CRISPR/Cas9 applications is crucial for advancing neuroimmune pharmacology and CNS disease treatment.
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