Parkinson: New gene therapy promises prevention

HealthScience

Signs of Parkinson’s disease and some forms of dementia including Lewy’s body, toxic aggregates that form in the brain and disrupt the chain of neurons. Researchers at Osaka University in Japan are now testing new preventive therapies in early rat studies.
According to information from the Parkinson Foundation, about 1 million people in the United States will contract Parkinson’s disease by 2020, and around 60,000 adults in the United States will be diagnosed with it every year.

More than 10 million people worldwide suffer from Parkinson’s disease. Although so extensive, scientists are still not sure where it came from, and doctors only prescribe symptomatic treatment to treat this condition.

Nevertheless, researchers continue to investigate the causes and possibilities of preventive therapy. Recently, a team of scientists from Osaka University in Japan discovered whether taking a protein called alpha-synuclein, which accumulates in Levi’s body, can help prevent or restore Parkinson’s disease.

For this purpose, they tested new gene therapy in mice with this neurological disease. Their conclusions, contained in scientific reports, show that this new approach is promising and that scientists must continue their research.

“Although there are drugs that treat symptoms associated with [Parkinson’s disease], there is no basic treatment to control the occurrence and development of the disease,” said lead author Takuya Wehara.

“Therefore, we have been looking for ways to prevent the expression of alpha-synucleins and to effectively eliminate the physiological causes of Parkinson’s disease,” Wehara added.

Consequences for Parkinson’s and dementia

Initially, the team “reflected” parts of genetic material that corresponded to those related to alpha synuclein. The researchers then used the amido bridge technique that uses amino radicals to bind molecules to stabilize these genetic fragments.

For this reason, they call the new genetic fragment of amide a modified antisense oligonucleotide or ASO bridge. This fragment binds to the appropriate genetic sequence, which is information RNA (mRNA). The role of mRNA is to “decipher” genetic information by translating it into protein.

By connecting to mRNA, ASO cannot translate genetic information encoding alpha-synuclein, a protein that forms Lewy’s body.
Researchers have experimented with various ASO variants until they found one which reduced the level of alpha-synuclein mRNA to 81%. Finally, the team tested the effectiveness of their new approach in the mouse model.

“When we tested ASO in a mouse model of [Parkinson’s disease], we found that it was sent to the brain without the need for a chemical carrier,” explained Chi-Jing Choong, lead author of the study. In mice, new gene therapy has proven effective and promising.

“Additional tests show that ASO effectively reduces alpha-synuclein production in mice and significantly reduces the severity of disease symptoms within 27 days of administration,” Choong said.

In the future, researchers want to continue testing this method. If efforts continue to prove successful, they hope that the new therapeutic approach will not only contribute to the prevention and treatment of Parkinson’s disease, but also to other neurodegenerative diseases in which Lewy’s body plays a key role.

Leave a Reply

Your email address will not be published. Required fields are marked *