Researchers from the University of Illinois Urbana-Champaign have introduced an exciting new gene-editing technique known as SPLICER.
This innovative method is designed to help cellular machinery navigate through portions of genes associated with various diseases.
In a promising application, SPLICER was used in a mouse model of Alzheimer’s disease, successfully reducing the production of amyloid-beta plaque precursors.
These compelling results were published in the journal Nature Communications, with bioengineering professor Pablo Perez-Pinera leading the study.
SPLICER: A New Approach to Gene Editing
SPLICER stands out with its increased efficiency over existing gene-editing techniques.
This breakthrough could pave the way for addressing a wide range of genetic disorders.
The technique relies on a concept called exon skipping, aimed at treating conditions that stem from mutations leading to the creation of misfolded or harmful proteins, such as Duchenne’s muscular dystrophy and Huntington’s disease.
To illustrate the role of DNA in cellular function, the researchers compared it to a recipe book filled with instructions, some of which may be unnecessary or confusing.
They pointed out that large segments of DNA don’t code for proteins and can contain irrelevant information—imagine a recipe directing the cook to follow a page filled with nonsense.
When faced with critical errors in a gene, direct corrections may not always be possible.
Instead, the focus shifts to sidestepping the problematic areas while maintaining the overall function of the gene.
Enhancements Over Traditional Techniques
Building on the well-known CRISPR-Cas9 gene-editing system, SPLICER offers significant enhancements.
Unlike the traditional CRISPR-Cas9 approach, which relies on exact DNA sequences for targeting, SPLICER utilizes advanced Cas9 enzymes.
These newer enzymes can engage with a wider array of genetic targets, including the gene associated with Alzheimer’s disease that was the focus of this research.
Another notable advantage of SPLICER is its capacity to skip the desired exon precisely, eliminating the risk of leftover undesired sequences.
Current exon-skipping methods sometimes stumble, failing to remove the entire problematic segment—much like skipping a page mid-sentence in a recipe can create confusion.
Future Prospects and Safety Considerations
While exon skipping does have its limitations—particularly for diseases that depend on functional proteins—this technique shows considerable promise in treating neurological disorders, including Alzheimer’s, Parkinson’s, and Huntington’s disease.
Moving forward, the researchers plan to further investigate the safety of this approach, ensuring that the skipping process does not produce harmful or destabilizing proteins.
Additionally, they aim to conduct long-term studies to observe disease progression after treatment.
Source: Science daily