Targeted Knockdown Of Human SOD1 Genes By Non-viral Gene Delivery To Delay Onset And Progression Of ALS
Funder
National Health and Medical Research Council
Funding Amount
$504,097.00
Summary
Amyotrophic lateral sclerosis (ALS) is an illness of nerves resulting in a creeping paralysis and death; there is no effective treatment. We have developed immunogenes consisting of an antibody to target specific nerves and a gene that can affect it. Our immunogene will deliver genes that inhibit a mutant protein causing disease in an ALS mouse model. Successful outcomes of this research will be to encourage development of treatments both before and after the disease has developed.