Effect of TTR Gene Therapy in Alzheimer's Disease

A protein called TTR will be studied to find out whether it can be used to treat people with Alzheimer’s Disease (AD). TTR has been shown to have an effect in mice who show symptoms like those of AD in humans. It is unknown whether TTR will have an effect on the disease in humans as well. Different types of TTR, where it is given, and when it is given will be studied.

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
in AD.

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.