Blood-Brain Barrier Penetrating Antisense Therapy For Spinal Muscular Atrophy

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/GNT1138033

Funding Status
Closed

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Funded Activity Summary

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.

Funded Activity Details

Start Date: 01-01-2018

End Date: 01-01-2020

Funding Scheme: Project Grants

Funding Amount: $635,005.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Basic Pharmacology

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

antisense oligonucleotides | blood-brain barrier | drug delivery systems | motor neuron disease (MND) | peptides

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