Summary

We have identified Ubiquitin homeostasis as a novel therapeutic target for the treatment of amyotrophic lateral sclerosis (ALS).

The Ubiquitin (Ub) system is severely compromised in ALS patients and we aim to restore capacity of this system via gene therapy.

Specifically, we will deliver the Ub gene to the nervous system (i.e. motor neurons) using a targeted, sterically stabilized liposome delivery system.

This proposal has two unique lines of innovation

  1.  a novel target, and
  2. a novel drug delivery system for motor neurons.

Both of which will have a significant impact on development of therapeutics for ALS.

Funding body

United States of America Department of Defence

Scheme name

Amyotrophic Lateral Sclerosis Research Program Therapeutic Idea Award

Years funded

2016 – 2018

Lead institution

University of Wollongong