SMN IMMUNOGENE THERAPY FOR MOTOR NEURON DISORDERS

Grant number: 1104299 | Funding period: 2016 - 2018

Completed

Abstract

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)

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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

2021-04-15

Antisense oligonucleotides (ASOs) are an emerging class of gene-specific therapeutics for diseases associated with the central ner..

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Amyotrophic Lateral Sclerosis and Autophagy: Dysfunction and Therapeutic Targeting

Azin Amin, Nirma D Perera, Philip M Beart, Bradley J Turner, Fazel Shabanpoor

2020-11-01

Over the past 20 years, there has been a drastically increased understanding of the genetic basis of Amyotrophic Lateral Sclerosis..

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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

2020-05-01

Motor neuron degeneration in amyotrophic lateral sclerosis (ALS) is proposed to occur by necroptosis, an inflammatory form of regu..

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Androgen receptor antagonism accelerates disease onset in the SOD1(G93A) mouse model of amyotrophic lateral sclerosis

Victoria M McLeod, Chew L Lau, Mathew DF Chiam, Thusitha W Rupasinghe, Ute Roessner, Elvan Djouma, Wah C Boon, Bradley J Turner

2019-07-01

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Thiol-Cyanobenzothiazole Ligation for the Efficient Preparation of Peptide-PNA Conjugates

Nitin A Patil, John A Karas, Bradley J Turner, Fazel Shabanpoor

2019-03-01

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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

2018-06-01

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Rilmenidine promotes MTOR-independent autophagy in the mutant SOD1 mouse model of amyotrophic lateral sclerosis without slowing disease progression

Nirma D Perera, Rebecca K Sheean, Chew L Lau, Yea Seul Shin, Philip M Beart, Malcolm K Horne, Bradley J Turner

2018-01-01

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The Assembly of Fluorescently Labeled Peptide-Oligonucleotide Conjugates via Orthogonal Ligation Strategies

John Karas, Bradley J Turner, Fazel Shabanpoor

2018-01-01

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Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts

Anna Farrelly-Rosch, Chew Ling Lau, Nitin Patil, Bradley J Turner, Fazel Shabanpoor

2017-09-01

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Emerging Therapies and Challenges in Spinal Muscular Atrophy

Michelle A Farrar, Susanna B Park, Steve Vucic, Kate A Carey, Bradley J Turner, Thomas H Gillingwater, Kathryn J Swoboda, Matthew C Kiernan

2017-03-01

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