SPLICER demonstrates greater efficiency than existing gene editing technologies by utilizing a method known as exon skipping, specifically targeting genes linked to diseases caused by misfolded proteins.

Researchers at the University of Illinois Urbana-Champaign have developed an innovative gene editing tool called SPLICER, designed to effectively skip parts of genes associated with various diseases.

While exon skipping may not be applicable to all genetic diseases, it shows significant promise for conditions such as Alzheimer's, Parkinson's, Huntington's disease, and Duchenne's muscular dystrophy.

The study, led by bioengineering professor Pablo Perez-Pinera, was published in the journal Nature Communications and received support from various organizations, including the National Institutes of Health and the Muscular Dystrophy Association.

Perez-Pinera likens the process of exon skipping to a cookbook analogy, where omitting erroneous parts of a recipe enables the successful creation of a functional dish, akin to producing a functional protein despite skipping faulty genetic instructions.

Overall, SPLICER represents a significant advancement in gene editing technology, offering a more efficient and precise method for addressing genetic diseases linked to misfolded proteins.

This tool enhances precision by editing both the beginning and ending sequences of exons, allowing for more accurate skipping of unwanted genetic material without leaving behind partial sequences that could disrupt protein function.

In experiments with a mouse model of Alzheimer's disease, SPLICER successfully reduced the targeted exon by 25% in the brains of the mice, achieving this without off-target effects and leading to a decrease in amyloid-beta production.

Looking ahead, the research team plans to assess the safety of removing targeted exons and conduct longer-term studies to monitor disease progression in animal models.

SPLICER improves upon traditional CRISPR-Cas9 technology by employing newer Cas9 enzymes that do not require specific DNA sequences, thus broadening the range of gene targets, including those associated with Alzheimer's.

Previous exon-skipping methods struggled to effectively target specific exons, but SPLICER combines newer base editors with dual splice editing to achieve superior results.