Gene Therapy Success for Brian Disease

Naturenews reports on another important success for gene therapy-- treatment for ALD (adrenoleukodystrophy), a rare, inherited metabolic disorder that afflicts young males.

ALD results in severe degeneration of the structure that is crucial for brain-cell function and most die before adolescence.

Here is a sample from the news story:

Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells.

The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now.

...."It's a huge advance," says Mark Kay, director of the Program in Human Gene Therapy at Stanford University School of Medicine in California. "If you look in general at the vectors we use for gene therapy, we've really come a long way. This is the first successful use of lentiviral vectors, and it gives me a lot more cautious optimism moving forward."

The research article of the study is published in Science (paper here).

Cheers,
Colin