Over the Barrier

Gene therapy could radically change our approach to treating disease, but it still has many hurdles to overcome. One major challenge is to efficiently deliver genes into target cells, especially in the brain. The blood-brain barrier, which shields the brain from pathogens in the bloodstream, is difficult to cross, even for the most promising gene vectors, small modified viruses known as adeno-associated viruses (AAVs). Researchers have recently developed a protocol for identifying the best AAVs to send into the brain, based on generating variants with different outer shells, or capsids, then screening for those that penetrate brain cells most easily. In this mouse cerebellum, green fluorescence demonstrates integration of a gene carried by the most effective AAV they generated. Applying this vector could enable more successful and less invasive treatment of the brain than current techniques, while the general method could help design improved AAVs for other tissues too.

Written by Emmanuelle Briolat

Emmanuelle Briolat

Writer

A researcher specialising in visual signals in Lepidoptera, camouflage and the effects of light pollution, Emmanuelle’s writing covers a range of topics, from biomedical research to children’s books about moth defences.

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• Image by Ben Deverman and the Gradinaru laboratory/Caltech
• Caltech Division of Biology and Biological Engineering, Pasadena, CA, USA
• Image copyright held by original authors
• Research published in Nature Biotechnology, February 2016