Researchers Use Cloned Gene To Cure 3-Year-Old "Bubble Baby"

PHILADELPHIA (CBS) --  Researchers say they've cured a disease that can be fatal in infants.

The treatment involves using a cloned gene.

Days after bringing home her newborn twin daughters, Alysia Vaccaro could sense Evangelina wasn't as healthy as her sister. "I had a baby to compare her to. I just knew something was wrong with her," she said.

Her mother's intuition was right.

Testing revealed Evangelina was born with Severe Combined Immunodeficiency – also called "skid" or "bubble baby" disease.

It's a genetic disorder that leaves the body without an immune system making infants extremely vulnerable to illness. Even the common cold can be deadly.

We wore masks, we had hand sanitizer. We had raw hands from cleaning so much," Vaccaro said.

The Vaccaro's turned to a clinical trial as a last hope.

And that lead to a cure.

The treatment takes bone marrow from the patient to gather stem cells.

A cloned gene is then added to correct what was missing at birth.

"Those stem cells are given back to the patient where they can go back to the bone marrow and make the blood cells for the rest of the patient's life," Dr. Donald Kohn, of UCLA's Broad Stem Cell Research Center said.

So far, the treatment has restored immune systems in all 23 patients in the most recent clinical trials.

That includes Evangelina.

"It is a cure. I know it's a cure. We're living the cure," her mother said.

Now three years old, doctors say she's in perfect health.

The clinical trial was performed at UCLA. Doctors there are now working with the FDA to make the treatment available nationwide. That very same method is now being tested for a cure of sickle cell disease.

Early diagnosis of skid is still unusual because it's not included in newborn testing.