Breaking through the barrier

A non-invasive method to deliver genes to the central nervous system in mice is reported online this week in Nature Biotechnology. The research could one day lead to new therapies for neurodegenerative disorders such as Lou Gehrig’s disease.

Getting drugs or genes into the brain and spinal cord to treat neurodegenerative diseases is problematic because of the blood?brain barrier (BBB)?a tight wall of cells that restricts the movement of molecules between the blood and neural tissue. Until now, no viruses or viral vectors have been found that cross through the BBB after intravascular injection. Brian Kaspar and colleagues show that a particular strain of virus ? AAV9 ? can be used to carry genes across the BBB in mice and into the brain cells beyond. The team also find that the virus targets cells of the spinal cord and so can be used to deliver genes to widespread regions of the central nervous system.

It is hoped that the technology could one day be used to introduce working copies of the genes that are damaged in diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease.

doi: 10.1038/nbt.1515

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