Can genetic diseases be cured?

(Lansing State Journal, March 9, 1994)

Because there are so many genetic diseases, there is no simple answer to that question. Lately, much attention has been given to developing gene therapies, because they offer new possibilities to alleviate human suffering. However, the nature of genetic disease makes them difficult to cure.

Certain diseases are caused by lesions in just a single gene. While a human cell may have nearly 100,000 genes, an error in just one can have severe repercussions on a person's whole physiology. One example is ADA, an immune system disorder caused by a deficiency in a single gene product.

Some diseases caused by a single aberrant gene, such as ADA, may be treatable with gene therapy. In fact, AVA was the first disease to be successfully treated using gene therapy. The technology exists to transfer a healthy gene into a patient's own cells, giving those cells the ability to recover.

However, not all single gene diseases can be treated this way. One difficulty lies in the fact that there are over ten trillion cells in a human being, and not all of them can be treated once the person is born.

Other diseases, such as Down syndrome, have more complex causes, and cannot be cured directly by introducing a single healthy gene into an affected cell.

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