Newly Developed Gene Editing Technology Decreases Alzheimer’s Plaque in Mice

Gene editing tools have been continually explored and improved for the past half-century as a way to gain a deeper understanding of genetic functions, as well as treat and prevent genetic diseases such as cystic fibrosis and diabetes. Recently, a team at the Univ. of Illinois Urbana-Champaign (UIUC) showed that their newly developed gene editing tool, called SPLICER, can be used to reduce the formation of amyloid-beta plaque precursors — proteins linked to Alzheimer’s disease — in mice.

Globally, nearly 55 million people are living with Alzheimer’s disease or other dementias, a number that is expected to increase to 139 million by 2050. Approximately 7 million of these are Americans, while about 60% are people living in low- and middle-income countries.

SPLICER has the opportunity to change this while also helping prevent other genetic conditions. The gene editing technology uses a ribonucleic acid (RNA) splicing approach called exon skipping, which involves encouraging cells to skip over faulty or misaligned genetic code sections, or exons, to create a modified yet functional protein.

“DNA contains the instructions to build everything that is responsible for how cells function,” says Pablo Perez-Pinera, UIUC bioengineering professor and the study’s principal investigator. “But there are...