

A new gene editing technique – SPLICER that allows cellular machinery to skip sections of genes involved for diseases has been used to minimize the creation of amyloid-beta plaque precursors in a mouse model of Alzheimer’s disease, according to researchers at the University of Illinois Urbana-Champaign.
The application in living mice demonstrates the technique, called SPLICER,’s improved efficiency over the current standard in gene editing technology, as well as its potential for use in other disorders, the researchers said. The study, led by Pablo Perez-Pinera, a bioengineering professor at the University of Illinois, was published in the journal Nature Communications.
SPLICER employs a gene editing technique known as exon skipping, which is particularly useful for health disorders caused by mutations that result in misfolded or toxic proteins, such as Duchenne’s muscular dystrophy or Huntington disease.
“DNA contains the instructions to build everything that is responsible for how cells function. So it’s like a book of recipes that contains very detailed instructions for cooking,” Perez-Pinera said.
But there are large regions of DNA that don’t code for anything. It’s like, you start the recipe for a turkey dinner, and then you hit a note that says, ‘continued on page 10.’ After page 10, it’s ‘continued on page 25.’ The pages between are gibberish.”
Pablo Perez-Pinera, Professor, Bioengineering, University of Illinois at Urbana-Champaign
“But say on one of the recipe pages -; in genetics, an exon -; there is a typo that makes the turkey inedible, or even poisonous. If we cannot correct the typo directly, we could amend the note before it to send you to the next page, skipping over the page with the error, so that at the end you could make an edible turkey. Though you might lose out on the gravy that was on the skipped page, you’d still have dinner. In the same way, if we can skip the piece of the gene with the toxic mutation, the resulting protein could still have enough function to perform its critical roles.”
SPLICER extends the popular CRISPR-Cas9 gene editing tool with significant improvements. CRISPR-Cas9 systems require a certain DNA sequence to latch onto, which limits the genes that can be altered. SPLICER employs newer Cas9 enzymes that do not require that sequence, opening the door to additional targets such as the Alzheimer’s-related gene that the Illinois researchers focused on.
“Another problem we address in our work is precision in what gets skipped,” said graduate student Angelo Miskalis, a co-first author of the paper. “With current exon-skipping techniques, sometimes not all of the exon gets skipped, so there’s still part of the sequence we don’t want expressed. In the cookbook analogy, it’s like trying to skip a page, but the new page starts in the middle of a sentence, and now the recipe doesn’t make sense. We wanted to prevent that.”
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