SMN IMMUNOGENE THERAPY FOR MOTOR NEURON DISORDERS
Grant number: 1104299 | Funding period: 2016 - 2018
We have designed novel gene therapy agents to treat MND. This therapeutic approach uniquely combines gene therapy agents with antibodies which stimulate motor neuron health and connections to muscle. This project will comprehensively characterise the therapeutic effects of our novel gene therapy agents in MND mice. We predict that our gene therapy complexes will improve motor neuron survival,motor function and lifespan in MND mice.
Related publications (10)
Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides.
Mohadeseh Dastpeyman, Ramin Sharifi, Azin Amin, John A Karas, Brittany Cuic, Yijun Pan, Joseph A Nicolazzo, Bradley J Turner, Fazel Shabanpoor
Antisense oligonucleotides (ASOs) are an emerging class of gene-specific therapeutics for diseases associated with the central ner..
Amyotrophic Lateral Sclerosis and Autophagy: Dysfunction and Therapeutic Targeting
Azin Amin, Nirma D Perera, Philip M Beart, Bradley J Turner, Fazel Shabanpoor
Over the past 20 years, there has been a drastically increased understanding of the genetic basis of Amyotrophic Lateral Sclerosis..
Necroptosis is dispensable for motor neuron degeneration in a mouse model of ALS
Taide Wang, Nirma D Perera, Mathew DF Chiam, Brittany Cuic, Nayomi Wanniarachchillage, Doris Tomas, Andre L Samson, Wayne Cawthorne, Eric N Valor, James M Murphy, Bradley J Turner
Motor neuron degeneration in amyotrophic lateral sclerosis (ALS) is proposed to occur by necroptosis, an inflammatory form of regu..
Association of Regulatory T-Cell Expansion With Progression of Amyotrophic Lateral Sclerosis A Study of Humans and a Transgenic Mouse Model
Rebecca K Sheean, Fiona C McKay, Erika Cretney, Christopher R Bye, Nirma D Perera, Doris Tomas, Richard A Weston, Karlene J Scheller, Elvan Djouma, Parvathi Menon, Stephen D Schibeci, Najwa Marmash, Justin J Yerbury, Stephen L Nutt, David R Booth, Graeme J Stewart, Mathew C Kiernan, Steve Vucic, Bradley J Turner