Investigating the potential of targeting resident neural stem cells in Ataxia Telangiectasia

Prog Paul Wong

Research Project Information

Principal researchers: Professor Paul Wong
Institute: MD Anderson Cancer Centre, University of Texas, USA
Project completion date: December 2013

Project Overview

Ataxia-telangiectasia (A-T) is caused by faults in a gene called ATM (Ataxia-Telangiectasia Mutated). Similar to Alzheimer’s and Parkinson’s disease, some of the most destructive symptoms are due to the death of nerve cells (neurons) within the brain. For instance, this neurodegeneration is responsible for the loss of muscle coordination (called ataxia) observed in A-T patients. Unfortunately, no current therapy can prevent progressive neurodegeneration and ataxia.

The use of neural stem cells (NSCs) may provide promising opportunities for treating A-T patients. NSCs have the remarkable potential to develop into neurons. It may therefore be possible to use NSCs to replace neurons that are lost as a result of A-T. The main goal of this project was to investigate the potential of using resident NSCs to reduce neurodegeneration in A-T patients.

Project Outcome

The cerebellum region of the brain plays an important role in muscle coordination. Consequently, the death of neurons within the cerebellum can cause ataxia. Professor Paul Wong and his research team transplanted NSCs into the cerebellum region of A-T mice’s brains. The research team report that the transplant procedure shows promise and merits further investigation.


As of January 2016, these results have not been published.

What Next?

Due to the retirement of Professor Paul Wong on the 31st August 2014, any further research in this area will now be carried out by Dr. Ashok Shetty.