Neuroscientists at Lund University have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The approach, called BRAVE (barcoded rational AAV vector evolution), allows for the efficient selection of engineered capsid structures on a large scale as well as improves the accuracy of therapy.
"Thanks to this technology, we can study millions of new virus variants in cell culture and animal models simultaneously. From this, we can subsequently create a computer simulation that constructs the most suitable virus shell for the chosen application—in this case, the dopamine-producing nerve cells for the treatment of Parkinson's disease," says Tomas Björklund, senior author of the paper published in PNAS earlier this week.
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With the new method, researchers have been able to significantly reduce the need for laboratory animals, as millions of variants of the same drug are studied in the same individual. They have also been able to move important parts of the study from animals to cell culture of human stem cells.
"We believe that the new synthetic virus we succeeded in creating would be very well suited for gene therapy for Parkinson's disease, for example, and we have high hopes that these virus vectors will be able to be put into clinical use,” adds Björklund.