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Updated: Dec 30, 2025

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
Published on: May 30, 2025
Genetic and epigenetic editing in nervous system .
1Author affiliations: Department of Neurobiology, University of Alabama at Birmingham, Alabama, US. Address for correspondence: Jeremy J. Day, PhD, Associate Professor, Department of Neurobiology, University of Alabama at Birmingham, 910 Shelby Building, 1825 University Blvd, Birmingham, AL 35294, US. (email: jjday@uab.edu).
Gene editing tools like CRISPR offer new ways to study and potentially treat brain disorders by precisely controlling gene expression in neurons. These technologies are advancing our understanding of neurological diseases linked to genetic and transcriptional dysregulation.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Neuronal functions rely on precise gene expression, crucial for brain health.
- Disruptions in gene regulation are implicated in neurodevelopmental, neuropsychiatric, and neurological diseases.
- Gene editing technologies are rapidly advancing the study of the central nervous system.
Purpose of the Study:
- To review recent advances in genetic editing, transcriptional modulation, and epigenetic reorganization.
- To highlight applications in neuronal systems and potential uses in brain disorders.
- To discuss the role of gene editing in understanding gene regulatory function and epigenetic modifications.
Main Methods:
- Review of recent literature on gene editing technologies, including CRISPR-based tools.
- Focus on applications for cell-specific modulation and temporal dynamics.
- Examination of epigenetic editing at specific gene loci.
Main Results:
- Gene editing tools enable rapid understanding of gene sequence variation and regulatory function in the CNS.
- CRISPR-based tools allow for cell-specific modulation and sophisticated interrogation of temporal dynamics.
- Epigenetic editing provides insights into locus-specific functions and disease contexts.
Conclusions:
- Advances in gene editing, transcriptional modulation, and epigenetic reorganization offer powerful tools for neuroscience.
- These technologies have significant potential for understanding and treating brain disorders.
- Future applications may target genetic variations and transcriptional dysregulation in neurological diseases.
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