Effect of TTR Gene Therapy in Alzheimer's Disease
Detailed Non-Technical Summary
The protein transthyretin (or TTR) has been shown to have an effect in mice who have symptoms like those of Alzheimer's Disease (AD) in humans, but it is unknown whether TTR will have an effect on the disease in patients as well. I am using adeno-associated virus (AAV), a safe and non-pathogenic type of virus, to deliver TTR to a mouse model of AD and study its potential effect on disease modulation. Our studies will investigate when and where expression of TTR can have a beneficial impact
If our hypothesis proves to be successful in halting disease progression, we will continue our line of research towards a better understanding of how TTR ameliorates AD progression beyond Aβ deposition and understanding the mechanistic relationship between the two proteins. These studies will be necessary to advance this approach to a potential clinical application.