The research group in Lund is led by Tomas Björklund, professor of neuroscience and head of the research group. His research focuses on developing new synthetic viruses that can safely and efficiently deliver genetic medicines to the correct nerve cells in the brain. Viruses have a unique ability to introduce new genetic material into cells—that is, the genetic blueprint that governs the formation of all the cell’s functional building blocks, proteins.
“Over the past few years, we have learned how to tame viruses so that they are safe and can deliver new treatments without spreading further. However, they are still relatively inefficient and poor at reaching the brain and the nerve cells that degenerate in Parkinson’s disease,” says Tomas Björklund.
Together with research colleagues at Karolinska Institutet, Björklund has developed a new technology that combines artificial intelligence and machine learning with advanced experiments in so-called organoids—mini-brains generated from human stem cells. Using this approach, the researchers are creating entirely new virus-like particles that are target-seeking and specifically directed toward dopamine neurons.
The hope is that these particles will eventually be administered to patients through a simple injection into the cerebrospinal fluid, where they could protect dopamine neurons from dying as a result of Parkinson’s disease.
Within the project, Thomas Perlmann’s research group at Karolinska Institutet identifies genes that can protect against Parkinson’s disease, while Karima Chergui’s group maps the mechanisms needed to deliver the newly identified protective genes. At the same time, Per Svenningson’s group works to identify which patients are most likely to benefit from the treatment and to develop the integrated therapy with the ultimate goal of reaching clinical application.
What does this grant mean for your research?
“This funding allows us to truly join forces and bring together several very strong ideas into a unified treatment strategy with great potential to reach the clinic. For our part, it is also crucial for recruiting a young researcher within the highly competitive field of AI and synthetic biology,” says Tomas Björklund.
A new gene therapy to slow Parkinson’s disease—how far have you come?
“So far, we have succeeded in creating a first generation of synthetic viruses that are far more effective than what was previously available. They infect dopamine neurons with very high specificity, but their current limitation is that they must be injected directly into the brain. If we can develop new variants that can be delivered via the cerebrospinal fluid, this would open the door for many more patients to be treated in the future,” Tomas Björklund concludes.