Researchers at Osaka University in Japan are testing a new preventive therapy for Parkinson’s disease. It is a preliminary mouse study evaluating the efficacy of the therapy in treating conditions like Parkinson’s. the finding of the study appeared in Scientific Reports.
Experts from Parkinson’s foundation estimated that around 1 million people in the U.S. will have Parkinson’s disease by 2020. Moreover, around 60,000 U.S. adults are diagnosed with this condition yearly. They further add that almost 10 million people are living with Parkinson’s across the world.
The cause of the condition is still poorly understood however; the hallmarks of the condition include Lewy bodies and toxic aggregates in the brain. These toxic reserves disrupt neural circuits affecting the cognitive functions and memory. Usually, doctors only prescribe symptomatic treatments for the management of this condition. Nevertheless, researchers continue to study its causes and possible preventive therapies.
A team of scientists from the University of Osaka in Japan recently carried out a study regarding the subject. They decided to find out whether targeting a protein called alpha-synuclein could prevent or reverse Parkinson’s disease. Note this protein aggregates into Lewy bodies. For this purpose, they have tested a new gene therapy in mice with a similar neurological condition. The research suggests that this new approach is promising and that scientists should take their investigations further.
According to medical experts, there is no basic treatment to control the onset and progression of Parkinson’s. however, there are certain drugs that treat the symptoms of the condition.
What did the research team do?
The researchers looked at ways to prevent the expression of alpha-synuclein and effectively eliminated the physiological cause of Parkinson’s disease. First of all, the team designed mirror sections of genetic material to match those that correspond to alpha-synuclein. Afterward, they used amido-bridging to stabilize these genetic fragments. Amido-bridging is a technique that employs amino radicals to connect molecules. This is the reason, the new genetic fragments were called amido-bridged nucleic acid-modified antisense oligonucleotides, or ASOs.
These fragments work by binding to their matching genetic sequence. This matching sequence is a messenger RNA (mRNA) that helps to decode genetic information, translating it into proteins. the binding of ASOs to mRNA prevents it from translating the genetic information, encoding alpha-synuclein, the Lewy body forming a protein.
The experiments continued with different ASO variants until the researchers found the one that lowered alpha-synuclein mRNA levels by as much as 81%. Finally, they tested the potential of their new approach in mouse models.
The results of the study report that the respective ASO was delivered to the brain without the need for a chemical carrier. Most importantly, it decreased alpha-synuclein production in the mice and significantly reduced the severity of disease symptoms within 27 days of administration. Thus, the novel gene therapy proved effective and promising in rodents.
The research team aims to continue testing this method and making efforts and improvements to prove it successful. They are hoping that the new therapeutic approach could help prevent and treat not just Parkinson’s disease but also other neurodegenerative conditions in which Lewy bodies play a crucial role.
The results of the study showed that gene therapy using alpha-synuclein-targeting ASOs is a promising strategy for the control and prevention of Parkinson’s disease. Therefore, they expect that in the future, this method will be used to not only successfully treat Parkinson’s disease, but also dementia caused by alpha-synuclein accumulation.