Blood-Brain Barrier Penetrating Antisense Therapy For Spinal Muscular Atrophy [ 2018 - 2020 ]

Research Grant

Researchers: Dr Fazel Shabanpoor (Principal investigator) ,  Dr Bradley Turner ,  Dr Joseph Nicolazzo

Brief description Spinal muscular atrophy (SMA) is a genetic disease caused by the deficiency of a protein known as survival motor neuron.This results in the degeneration of motor neurons (nerve cells controlling muscles) leading to progressive muscle weakness, paralysis, and eventual death. Currently, there is no known cure for SMA. The aim of proposed research is to develop gene-modifying molecules that prevent degeneration of motor neuron and extend the life-span of mice as a potential therapy for SMA.

Funding Amount $AUD 643,895.07

Funding Scheme Project Grants

Notes Standard Project Grant