Brain cells aglow after viral delivery

Newly identified virus could help replace faulty genes in human cells

cross-section of a mouse brain

PACKAGE DELIVERED  The whitish dots in this micrograph are neurons called Purkinje cells in the cerebellum of a mouse. Their color is due to a gene the cells received from the virus AAV-PHP.B.

B. Deverman and the Gradinaru laboratory/Caltech

In a multivirus competition, a newcomer came out on top for its ability to transport genetic cargo to a mouse’s brain cells. The engineered virus AAV-PHP.B was best at delivering a gene that instructed Purkinje cells, the dots in the micrograph above, to take on a whitish glow. Unaffected surrounding cells in the mouse cerebellum look blue. Cargo carried by viruses like AAV-PHP.B could one day replace faulty genes in the brains of people.

AAV-PHP.B beat out other viruses including a similar one called AAV9, which is already used to get genes into the brains of mice. Genes delivered by AAV-PHP.B also showed up in the spinal cord, retina and elsewhere in the body, Benjamin Deverman of Caltech and colleagues report in the February Nature Biotechnology.

Similar competitions could uncover viruses with the ability to deliver genes to specific types of cells, the researchers write. Selective viruses that can also get into the brain would enable deeper studies of the brain and might improve gene therapy techniques in people. 

BRIGHT BRAIN A mouse brain glows green due to the delivery of a gene by the virus AAV-PHP.B. B. Deverman and the Gradinaru laboratory/Caltech

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