Special Delivery (Destination: the brain)

Sep 2, 2003

Seven children, a fifty-minute drip, and a thimbleful of genetic elixir: such are the elements of a gene therapy trial that could lead to longer lives for children with the rare Canavan disease.

It all hinges on the work of Jude Samulski, a Carolina scientist who has over the last twenty years tailored a virus that swiftly but harmlessly sneaks into human cells and becomes one with DNA. His lab has fine-tuned adeno-associated virus (AAV) to be a “viral vector,” a minute delivery vehicle that will swap good genes for bad. Conceivably, this gene therapy can allow the body to stop and even reverse the effects of diseases such as muscular dystrophy, epilepsy, and the neurodegenerative Canavan disease.

Samulski has devoted his career to mastering AAV. (See Endeavors, Fall 1998, Star Bright, Cell Deep.) With a current Canavan gene therapy trial and one for muscular dystrophy on the way, his commitment to AAV is paying off: labs around the world now use Samulski’s viral vector.

Adeno-associated virus is a non-pathogenic parvovirus discovered as a contaminant in laboratory stocks of adenovirus, itself a candidate for use as a gene therapy viral vector. Because it can’t replicate without genes from “helper” viruses, and because it’s partial to a specific place in human chromosomes, AAV seems heaven-sent to be a viral vector.

And that’s what the parents of those seven children prayed for this summer.

... Read more at http://research.unc.edu/endeavors/fall2003/canavan.html

Endeavors Magazine, Fall 2003

Anton Zuiker

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