COIMBRA, Portugal, Sept. 3 (Xinhua) -- A new approach to treating Machado-Joseph disease has been developed by Portuguese researchers, it was announced this week.
Machado-Joseph disease is a hereditary neurodegenerative disorder with a high prevalence in parts of Portugal. Also known as spinocerebellar ataxia type 3, the disease causes progressive problems with movement, speech and swallowing. There is currently no cure for the disease.
The study, conducted by researchers at the University of Coimbra and the GeneT Center of Excellence in Gene Therapy and published in the scientific journal Biomaterials, could contribute to new treatments for the disease.
The researchers used tiny particles naturally released by cells to carry the CRISPR-Cas9 gene-editing tool, allowing them to target the mutated gene responsible for the disease.
"We wanted to understand whether it was possible to use extracellular vesicles to achieve transient exposure to the gene-editing machinery and minimize unwanted edits," said Kevin Leandro, first author of the study.
According to the researchers, the method could overcome some limitations of conventional gene therapies by reducing immune reactions and limiting the time gene-editing tools remain active in cells.
The researchers also developed a light-controlled system to improve the efficiency of the gene-editing process.
The University of Coimbra said the study is among the first internationally to demonstrate the effectiveness of this approach in Machado-Joseph disease, and could contribute to the development of safer treatments for the disease and other rare genetic disorders. ■