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Archive - Jun 7, 2019

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Novel Gene Therapy--Japanese Researchers Develop Modified Anti-Sense Oligo (ASO) for Possible Treatment & Prevention of Parkinson’s Disease; ASO Is Targeted Against mRNA for α-Synuclein

An Osaka University-led research team has recently published findings that provide a ray of hope for the millions of Parkinson's disease (PD) sufferers worldwide. Although more common in those aged over sixty, PD can strike at any age, with an estimated prevalence of 41 per 100,000 people in their forties. And, while not fatal in and of itself, the progressive neurodegeneration that is characteristic of PD can often cause secondary effects that lead to death. The exact cause of PD is still a mystery, but researchers believe that both genetics and the environment are likely to play a part. Importantly though, all PD patients show a loss of dopaminergic neurons in the brain and increased levels of a protein called α-synuclein (image), which accumulates in Lewy bodies. Lewy bodies are a pathological feature of both familial and sporadic forms of the disease, as well as some types of dementia. In the study published online on May 21, 2019 in Scientific Reports, the team led by researchers from Osaka University's Graduate School of Medicine focused on α-synuclein as a target for a novel PD treatment. The open-access article is titled "Amido-Bridged Nucleic Acid (AmNA)-Modified Antisense Oligonucleotides Targeting α-Synuclein As A Novel Therapy for Parkinson's Disease.” "Although there are drugs that treat the symptoms associated with PD, there is no fundamental treatment to control the onset and progression of the disease," explains lead author Takuya Uehara, PhD. "Therefore, we looked at ways to prevent the expression of α-synuclein and effectively eliminate the physiological cause of PD." To do this, the researchers designed short fragments of DNA that are mirror images of sections of the α-synuclein gene mRNA. The constructs were stabilized by the addition of amido-bridging.