What to Lego and Gene Therapy Have in Common?

Scientists have been using Adeno-associated viruses (AAVs) as a gene therapy vector for a number of years. Depite the fact that there are over 80 clinical trials that involve the use of AAVs worldwide (Wikipedia), AAVs lack the stability and specificity to deliver a gene to a specific target such as particular subregions of the brain.

In a new study taking place at Rice University, researchers have made use of computational and bioengineering methods to create new, benign viruses that can deliver DNA payloads to specific cells.

Their technique is premised upon an algorithm that predicts how parts of very large viruses can recombine by homing in on the viral protein sequences that work well together. According to senior scientist Jonathan Silberg, the researchers are using a hybrid approach to design and select the ideal mix of viruses which will deliver its gene payload in the most efficient manner.

“We’re treating them like Legos,” Silberg said. “We’re taking distantly related viruses that nature might not recombine very efficiently and looking for self-contained pieces of these proteins that can be swapped.”

To read more about this story see Rice writes rules for gene-therapy vectors.

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