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New therapy for 'Bubble Boy' disease - WCAX.COM Local Vermont News, Weather and Sports-

New therapy for 'Bubble Boy' disease

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LOS ANGELES -

The Ainslie family has been dealing with a rare disease since their son was born.

"He started getting really bad cough, vomiting, and throwing up," mom Jessica said.

Five-year-old Colton has severe combined immunodeficiency or SCID, also known as "Bubble Boy Disease." He's missing a gene needed for his immune system to develop. Kids with the condition are extremely vulnerable to viruses and bacteria and can die within one to two years if not treated.

Until now, a bone marrow transplant or lifelong enzyme injections were the only options. But now UCLA researchers are using an experimental gene therapy with low-dose chemotherapy.

"So take some bone marrow from the SCID baby and then in the laboratory add back to it a normal copy of the gene that they're missing that's causing SCID and then transplanting them back with their own bone marrow," said Dr. Donald Kohn of UCLA.

Doctors estimate just 30 to 40 kids are born every year in the U.S. with SCID.

There was a one in four chance Colton's little sister Abbygail would be born with the genetic disease.

"We knew right away. They did a blood test as soon as she was born," dad Michael said.

She had gene therapy treatment at just a few months old. Her parents say you'd never know the 14-month-old was sick.

"She's definitely doing a lot better than he is because we caught her so young," Jessica said.

Colton just started kindergarten.

"We're able to do whatever we want to do. We're not isolated in the house anymore," Michael said.

The Ainslies hope this experimental treatment will help their children keep living normal lives.

At this point, both children still need to take medications and have IV treatments to replace their antibodies to help fight infection.

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