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Special Issue

Antisense Oligonucleotide Therapeutics for Neurodegenerative Diseases

Submission Deadline: 28 Feb 2023

Guest Editors

  • Portrait of Guest Editor Dunhui  Li

    Dunhui Li PhD

    Perron Institute for Neurological and Translational Science, University of Western Australia, Perth, WA, Australia

    Interests: molecular medicine; antisense therapeutics; neuroscience; PTEN-induced Putative Kinase; Parkin Protein; Protein Deglycase DJ-1; Alpha Synuclein; Lewy Bodies; Parkinson DiseaseMachado-Joseph Disease; Spinocerebellar Ataxias; Polyglutamine

  • Portrait of Guest Editor Craig  McIntosh

    Craig McIntosh PhD

    Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Perth, Western Australia, Australia

    Interests: neurodegenerative; neuromuscular diseases

Special Issue Information

Dear colleagues,

The field of therapeutic antisense oligonucleotides (ASOs) is advancing very rapidly. In recent years, several ASO drugs have been approved by regulatory agencies including the US Food and Drug Administration and the European Medicines Agency for the treatment of different conditions. Five ASO compounds have so far been approved for neurological disorders, including Duchenne muscular dystrophy and spinal muscular atrophy. The success obtained with these two diseases has highlighted the potential of ASO therapeutics and stimulated interest from academia and industry to further develop this approach for other neurodegenerative diseases, including motor neuron disease, Huntington’s disease, Parkinson’s disease, Alzheimer’s disease, and spinocerebellar ataxias. Several ASO drugs designed specifically for these neurodegenerative diseases are currently under evaluation in different stages of clinical trials, with some showing promising outcomes.

For this Special Issue, “Antisense oligonucleotide therapeutics for neurodegenerative diseases”, we invite colleagues to submit original research articles, reviews, viewpoints, and research highlight papers on ASO therapeutics for neurodegenerative diseases. These may include, but are not limited to the following topics:

(1) The development of antisense oligonucleotide therapeutics for neurodegenerative diseases. 
(2) Novel antisense chemistries and chemical modifications with therapeutic potential for neurodegenerative diseases and other CNS conditions.
(3) Novel delivery approaches to help ASOs cross the blood-brain barrier.
(4) Novel combination treatments to enhance the efficacy of ASO.

Dr. Dunhui Li and Dr. Craig McIntosh 
Guest Editors

Keywords

  • antisense oligonucleotides
  • antisense chemistry
  • antisense delivery
  • neurodegenerative diseases

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