Spinal motor neurons (left) and astrocytes (right).

Rickie Patani : Human Stem Cells and Neurodegeneration Laboratory 

We study diseases of the nervous system, focusing on motor neuron disease (ALS) and dementia.

In ALS, patients lose the ability to move, eat, speak and ultimately breathe. ALS is untreatable because we do not understand the underlying cause(s) of disease.

In order to understand disease mechanisms, we use human stem cells generated from real patients. With over a decade of experience using this technology, we can now transform stem cells from patients into human nerve cells and, separately, their support cells (called glia). This approach allows us to determine the sequence of disease-related events within particular cell types. 

Our overarching goal is to identify precisely what goes wrong, when this begins and in which cell type. We specifically focus on how the following three factors contribute to nerve cell death in ALS:

  • Messages called RNAs, which are made from our DNA blueprint
  • Astrocytes, which are star-shaped cells that normally support nerve cells
  • Ageing, which is the biggest risk factor for many neurodegenerative diseases including ALS

The more we understand about human neurological diseases using this approach, the more we will be able to therapeutically target underlying disease mechanisms. We ultimately wish to use this new information to benefit patients with untreatable neurological diseases.