Sunday, July 30, 2023

Gene-Editing Tools Pave Way for New Alzheimer's Treatments

Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR, is a potent technique that enables precise gene modification. The research discussed centers on identifying individual genes linked to Alzheimer's disease in an effort to lower risk or decrease the illness's course.

Amyloid precursor protein (APP), which is essential for Alzheimer's disease: Depending on how it is broken down in the brain, the APP can produce metabolites that are either protective or pathogenic. In order to decrease the creation of beta-amyloid plaques, which are harmful protein deposits linked to Alzheimer's disease, while improving neuroprotective activities, the researchers employed CRISPR to alter the APP gene. In an Alzheimer's disease animal model, the CRISPR therapy resulted in a decrease in beta-amyloid plaques and related signs of brain inflammation as well as an increase in neuroprotective APP molecules. 

The APOE-e4 gene, which is regarded as a prevalent risk gene for Alzheimer's, is the focal point of the additional investigation. Having two copies of APOE-e4 raises risk of Alzheimer's disease by up to 12 times, whereas inheriting one copy increases risk by two to three times. In humanized mouse models and small brains made from Alzheimer's patients, the study demonstrated that levels of APOE-e4 may be greatly lowered using CRISPR without altering levels of other APOE variations that are thought to be neutral or protective. This strategy has the potential to help cure or stop Alzheimer's disease.

1 comment:

  1. Looking at the structures of the pathogenic APOE-4 compared to the protective APOE-2 and APOE-3 , it is surprising to see how slight the difference really is structurally. I wonder if a mutation was the origin of APOE-4.