Gene Therapy Gets A Good Rap

I read a story this weekend which made me absolutely glad that I've been a scientist.

"The biggest risk is she could die," Walter said. "And she was going to die anyway."

Who is Walter Coover talking about? His three month old daughter, Ariel.

Ariel has (had?) a genetic disorder called Canavan's disease. It's effect is atrophy of the brain. The Coovers are intimately familiar with this disease - their first baby, Amber, would have been 13 this year.

When Ariel tested positive in utero, the Coovers made an appointment with an abortion clinic. Something stopped them.

After she was born, they found out about this gene therapy research. Their baby is a guinea pig, but, as Walter said, the biggest risk is that she would die, which she was destined to do. And the gene therapy has been showing significant promise. All of the other patients (10 of them) have demonstrated complete cessation of atrophy. Of course, the damage already done can not be fixed.

In Ariel's case, though, no damage has occured yet.

Keep her in your thoughts and prayers. This little girl and her parents deserve a chance.

(*)>