Gene therapy rises again

After being used against two very rare diseases, gene therapy now holds the promise of much wider use.
Written by Dana Blankenhorn, Inactive

After several years of frustrating failure, gene therapy is rising again.

In theory gene therapy means finding a way to change a person's genetic structure so that an inherited condition may be reversed. In practice, it can mean taking a leukemia or HIV virus, altering its genetic structure, and hoping the cure doesn't turn out to be worse than the disease.

In the latest cases French scientists arrested the progress of ALD, the disease made famous by the movie "Lorenzo's Oil," while American scientists showed the potential of gene therapy to reverse a disease causing blindness.

In the French case, detailed at Science, scientists removed blood cells and treated them with a modified HIV virus carrying the gene for the enzyme the patients lacked. Chemotherapy wiped out the patients' blood marrow, the new cells were infused, and in time these cells began cranking out the needed protein.

It took over a year for evidence to suggest success -- one patient lost some vision, the other some points on a non-verbal IQ test -- but the general course of the disease had been halted.

The European Leukodystrophies Association (ELA), which has sponsored this research to the tune of 7 million Euros (almost $11 million) was over the moon.

Shown above is one of the two French patients alongside the group's "ambassador," former soccer star Zinedine Zidane. (He's the Frenchman who spat at the Italian rival during the World Cup final. Yeah, that guy. He's usually very nice.)

The story is heartwarming but the implications are far greater. Many common diseases like cancer are being shown to have a genetic component. As the use of gene therapy increases, and becomes regularized, we could be finding cures that can be used on millions.

That is at least a decade away, but now we know it's possible. We have a method.

This post was originally published on Smartplanet.com

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