Validating a novel gene therapy to treat diseases of the brain

Overview

This project is a continuation of work we have conducted over the past two decades that has investigated the physiological and potential therapeutic properties of a brain-derived protein known as soluble amyloid precursor protein-alpha (sAPPα). The project aim is to examine a potential sAPPα gene therapy to treat a form of dementia known as frontotemporal dementia (FTD).

We will use a viral vector to deliver the gene for human sAPPα to the brains of mice modelling FTD and use behavioural, electrophysiological and immunofluorescence tests to determine if the resulting over-expression of sAPPα prevents the development of memory deficits, deficits in neuronal function (synaptic plasticity, etc) and the neuropathology associated with the disease.

A second component to the project is to validate the passage of a highly novel viral vector across the blood-brain barrier (BBB). This vector binds to a human transporter protein found in the BBB, and that is expressed in our FTD mouse model, which then transports it into the brain where the genetic cargo is released. This project offers the opportunity to be involved in an exciting and novel gene therapy project and to learn a variety of cutting-edge and standard laboratory neurological techniques.

Honours and masters' positions available.

Useful information

• Graduate research at the University of Otago
• Scholarships at the University of Otago