Our research team has developed a non-invasive brain-wide genome-editing system for the treatment of familial Alzheimer’s disease. This groundbreaking technology enables the modification of the genetic causes of the disease throughout the brain.
The system has been tested in genetically modified mouse models, demonstrating promising results in reducing Alzheimer’s disease pathologies across the brain, accompanied by an improved cognitive function.
The system can be administered via a single non-invasive intravenous injection, effectively crossing the blood-brain barrier and delivering the genome-editing tool to the brain. The effects of this treatment can last a lifetime, presenting a potential “once-and-for-all” solution for patients with familial Alzheimer’s disease.
This work marks an important milestone in the field of genome editing for the treatment of hereditary brain diseases. Additionally, it contributes to the advancement of precision medicine for other inherited forms of neurodegenerative diseases.