AIDS virus used to help immunodeficient babies, toddlers

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An experimental therapy containing the AIDS virus helped to develop an immune system in 48 babies born with an immunodeficiency, according to a report from The Associated Press.

Fifty children who were born without an immune system were given an experimental gene therapy that included the AIDS virus, according to the AP. Of the 50 participants, all but two now have the ability to fight germs.

Donald Kohn, a doctor at the UCLA Mattel Children’s Hospital and the study’s leader, said the children who responded positively to the therapy are now “basically ‘free range’ — going to school, doing normal things” without the fear that an infection could turn life-threatening, the wire service reported.

“We’re taking what otherwise would have been a fatal disease” and helping these ill children with a single treatment, Kohn added.

The two patients who did not benefit from the gene therapy underwent successful bone marrow transplants later on.

Doctors, according to the AP, said it will take more time to determine if any of the 50 participants are cured, but they seem to be doing well so far.

The children administered the therapy had severe combined immunodeficiency syndrome, known as SCID, according to the wire service. The illness is caused by an inherited genetic flaw and prevents the bone marrow from creating healthy versions of the blood cells that develop immunity. 

The syndrome is known as “bubble boy disease” because of a 1970s case involving a Texas boy who lived in a plastic bubble for 12 years to protect himself from germs. 

Now, however, it is referred to as “bubble baby disease,” because approximately 20 different gene defects, including some that affect both males and females, can cause the illness, the wire service noted.

According to the AP, people with the illness usually die within their first two years of life without treatment.

Tags Gene therapy Rare diseases

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