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A Lactose Intolerance Cure!?

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You may have heard rumors swirling about a cure for Lactose Intolerance (LI). The rumors are true. The cure isn't. Now sit down and take a deep breath while I give you the facts at hand.

First, some quick background. All humans have a gene on their chromosone 2. This gene regulates the manufacture of an enzyme called lactase. Lactase breaks down, or digests, the milk sugar that is called lactose.

For most of the world's population, this gene tells the body to stop manufacturing lactase at some point in their lives.

For other people, however, this gene never sends out that signal. They can drink milk all their lives.

The lactase itself is actually manufactured in the small intestine, at the ends of fingertip-like projections called villi.

OK, got that?

Now switch to the Central Nervous System Gene Therapy Center at Thomas Jefferson University in Philadelphia, where neuroscientist Matthew During, M.D., Professor of Neurosurgery and Center Director, is doing gene therapy research.

Dr. During's idea is that he can put genes into a specially modified virus and have that virus enter the cells where they would do the most good, in this case intestinal cells. In this way the gene would override the body's own gene. Not only that, he thought that the virus would work just by letting it be swallowed in a liquid.

He used rats in the experiment, of course, not humans. But it all worked. The gene attached itself to the intestinal cells where lactase is made. Not in all the cells – just in about 20% of them – but enough to call the experiment a success, since it worked in every rat.

He wasn't looking for a cure for LI; the condition happened to work to test out his scheme. He's really more concerned about diseases such as diabetes. Not too surprising, since the work was supported in part by the Juvenile Diabetes Foundation International.

And the work is all terribly preliminary. I would guess it would be years before human diseases could be treated in this way, and even longer before it becomes a common procedure. And who knows if anyone will bother to do the procedure for LI at all?

Said Eric W.F.W. Alton of England's National Heart and Lung, Institute, "[It's] rather unlikely that gene therapy will be widely used for treating lactose intolerance."

But another report quoted Dr. During as saying, "The possible lactose treatment had been licensed to a commercial developer, and the team was now moving onto widening the potential application of the breakthrough to other genetic disorders."

So who knows? Diabetes and other far more serious diseases will have first priority, I'm sure.

Still, it would be nice, wouldn't it? Anybody out there with a million dollars and a dream?

The full report on the experiment can be found in the October 1998 issue of the science journal Nature Medicine.

During, Matthew J et al. "Peroral gene therapy of lactose intolerance using an adeno-associated virus vector." Nature Medicine 1998;4(10):1131-1135.

also see Alton, Eric WFW, Griesenbach, Uta & Geddes, Duncan M. "Milking gene therapy". (News & Views) Nature Medicine 1998;4(10):1121-1122.


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