The potential for developing molecular therapies for the treatment of Parkinson’s Disease is explored in a recent paper by researchers and clinicians from Western Australia’s Perron Institute, the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT) at Murdoch University and international collaborators.
The viewpoint paper is the first to focus on the prospects for applying rapidly advancing antisense strategies to target specific disease-causing aspects of Parkinson’s with RNA-based therapeutics that could be applicable to both hereditary and sporadic forms.
Entitled: ‘Targeted Molecular Therapeutics for Parkinson’s Disease: A Role for Antisense Oligonucleotides?‘, it was published in Movement Disorders – a highly prestigious journal in the Parkinson’s field.
The Lead Author is postdoctoral researcher Dr Dunhui (Oliver) Li (MD, PhD) from the Motor Neurone Disease Genetics and Therapeutics Research group. This group, led by Senior Author, Professor Anthony Akkari (Perron Institute and CMMIT), aims to identify genetic mechanisms and mutations in neurological conditions and to develop personalised treatments based on antisense oligonucleotide (AO) technology.
“Parkinson’s disease is a common neurodegenerative disease characterised by marked diversity in clinical symptoms and complex genetic background,” Dr Li said.
“The current view is that rather than representing a singular disorder, Parkinson’s encompasses a group of distinct biological entities with overlapping clinical signs and symptoms.
“Emerging efforts focusing on developing disease-modifying therapies have been hampered by this wide variation in symptoms and the molecular complexity of Parkinson’s, highlighting the importance of targeted therapeutic approaches for the disease.
“The rapidly developing field of AO therapeutics is a powerful approach which can be used to manipulate gene expression in many ways.
“Our recently published paper is the first article that emphasises targeted molecular therapy for Parkinson’s patients arising from mutations in the PARK2 gene using the AO approach to gene therapy.”
Last Author Professor Akkari said: “Most of the current efforts seeking to develop disease-modifying treatments for Parkinson’s have been focusing on the common disease-causing features, but alternative approaches are urgently needed for this devastating disorder.
“This paper provides new insights on the potential for antisense therapeutics for people with Parkinson’s, helping to pave the way for the development of innovative and targeted molecular genetic treatments.”
“It is a dream of mine to publish in the Movement Disorders journal,” Dr Li said. “Also, I’m very happy to collaborate with Professor Shengdi Chen at Shanghai Jiao Tong University, where I first started my research in developing innovative therapeutics in 2013.”
The other authors of the paper are Professor Frank Mastaglia AM (Perron Institute senior advisor and neurologist), Professor Steve Wilton AO (Perron Institute and CMMIT Director) and Dr Wai Yan (Wayne) Yau (Perron Institute consultant neurologist).
Funding support was provided by the Perron Institute and the Giumelli family.
Media contact: Tennille Kroemer, Perron Institute, mobile 0426 044 223