✍️ Author: Dr Eleni Christoforidou
🕒 Approximate reading time: 4 minutes
In the ever-evolving field of neuroscience, the CRISPR/Cas9 gene-editing system has been nothing short of revolutionary. This innovative tool allows scientists to make precise alterations to DNA sequences, and thus offers significant potential for understanding and treating neurological disorders.
CRISPR/Cas9, which stands for Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9, is a system adapted from a naturally occurring genome editing system in bacteria.
Here’s a simplified explanation of how it works:
The CRISPR sequence is a short RNA sequence that matches the DNA sequence to be edited.
The Cas9 enzyme acts like a pair of molecular scissors, cutting the DNA at the location specified by the CRISPR sequence.
The cell’s natural DNA repair machinery recognises the cut and repairs the DNA, either by sticking the ends back together, or by inserting a new piece of DNA at the cut site.
CRISPR/Cas9 has many potential applications in neuroscience:
Understanding Neurological Disorders: By creating animal models with specific genetic mutations associated with neurological disorders, scientists can study the disease mechanisms in a controlled setting.
Identifying Therapeutic Targets: Gene-editing with CRISPR/Cas9 can help identify the function of specific genes and their products, potentially uncovering novel therapeutic targets for drug development.
Developing Gene Therapies: In theory, CRISPR/Cas9 could be used to correct disease-causing mutations in the brains of people with genetic neurological disorders.
Gene editing using the CRISPR/Cas9 system has already been instrumental in advancing our understanding of neurodegenerative diseases:
Modelling Diseases: Scientists have used CRISPR/Cas9 to create animal models of diseases like Alzheimer’s, Parkinson’s, and ALS, providing valuable insights into these diseases.
Unveiling Disease Mechanisms: For example, scientists have used CRISPR/Cas9 to understand how mutations in the C9orf72 gene cause ALS and frontotemporal dementia.
Potential Therapeutic Application: Preliminary studies suggest CRISPR/Cas9 could be used to reduce the levels of disease-causing proteins in mouse models of neurodegenerative diseases.
CRISPR/Cas9 is a powerful tool that is revolutionising our approach to studying and potentially treating neurological disorders. While there are still technical and ethical challenges to overcome before it can be widely applied in a clinical setting, the potential of this gene-editing technology in neuroscience is truly exciting.