When Jessica Ainslie had her son Colton, she knew there was something wrong. Born prematurely, he weighed just 2 pounds, 9 ounces at birth, and he had to spend time in the neonatal intensive care unit before he could come home.

Thought to be out out of the woods, Colton then started developing breathing problems and was vomiting often, just a few months later.

It just kept getting worse. He wasnt eating a whole lot, and he couldnt keep anything down, Ainslie, from Peoria, Ariz., told FoxNews.com. We took him to the ER a couple times; they kept giving him fluids and sending him back home. But I knew there was something wrong with my kid. I had that mom gut feeling.

Multiple blood tests were done on Colton, but nothing definitive came up. Finally, one of Ainslies doctors said she was going to test for SCID, a possible diagnosis that made the new mom incredibly anxious.

For small children with severe combined immunodeficiency (SCID) more famously known as bubble boy syndrome disease and infection are a constant source of worry due to a weakened, or almost non-existent immune system. If left untreated, the condition is fatal in just one to two years.

Sure enough, Coltons blood work showed that he had ADA-deficient SCID, a form of SCID that affects 15 percent of SCID patients. While some treatments exist for those suffering from the disease, options are mostly limited to rare bone marrow matches or frequent drug injections that can last the entirety of the childs life. Though helpful, these treatments dont always work and can come with terrible side effects and high costs.

Devastated by the diagnosis, Ainslie was offered some hope when she was asked if they could Air Evac 10-month-old Colton to Dr. Donald Kohns office at UCLA, one of the leading doctors in ADA-deficient SCID. Now, after having participated in a new gene therapy study developed by Kohn, Colton and eventually his baby sister Abbygail are living normal lives, nearly SCID-free.

Be their own donor

Just about the time Colton was diagnosed, Kohn had been working on a novel gene therapy treatment for children with SCID. In collaboration with researchers from both the University of California at Los Angeles and that National Institute of Health, Kohn and his colleagues have spent 11 years creating and testing this new therapy, which is capable of restoring immune systems in children with ADA-deficient SCID.

What were doing with gene therapy is having the patient be their own donor, study author Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, told FoxNews.com. We insert the gene theyre missing and transport it back through their system.

Read more:
New Therapy for Bubble Babies

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