Could CRISPR gene therapy help treat Alzheimer’s disease?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene editing system, is a method scientists use to edit genes. CRISPR is like a pair of tiny molecular scissors that make a targeted cut in a specific location of a DNA sequence.

Once the DNA is cut, scientists can replace faulty genes with a healthy one, remove problematic genes or even introduce new genes altogether.

CRISPR has the potential to help us understand genetic diseases better and develop new treatments or help expedite the identification of drug targets, ultimately accelerating the drug discovery process.

Recently, scientists unveiled two new CRISPR-based therapeutic approaches to treat and prevent Alzheimer’s at the Alzheimer’s Association International Conference (AAIC) 2023, held in Amsterdam.

Decreased amyloid beta production after CRISPR

As part of the first study, researchers at the University of California San Diego developed a gene-editing technique using CRISPR that specifically targets the amyloid precursor protein (APP), which plays a critical role in Alzheimer’s disease.

The APP gene produces different products, some of which are protective (sAPPa), while others, like beta-amyloid, are associated with pathology.

This approach aims to decrease the production of beta-amyloid while promoting neuroprotective actions. To test their strategy, the researchers conducted experiments on mice with Alzheimer’s disease.

They found that CRISPR treatment resulted in a reduction in beta-amyloid plaques, a decrease in brain inflammation markers, an increase in neuroprotective APP products, and improvements in behavioral and nervous system function.

Importantly, CRISPR editing did not cause any undesirable side effects in healthy mice.

Dr. Brent Aulston, lead author and postdoctoral scholar at the Altman Clinical and Translational Research Institute at UC San Diego, told Medical News Today that “the idea of our therapeutic is to utilize CRISPR to introduce a change in the patient’s genome that is protective against Alzheimer’s disease.”