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UCLA lab researches new potential treatment for bubble baby disease

By Xinchen Li

April 12, 2017 11:33 p.m.

A UCLA research team has developed a new stem cell gene therapy to treat babies with diminished immune systems.

Adenosine deaminase-deficient severe combined immunodeficiency, or bubble baby disease, is an immune system defect caused by the lack of adenosine deaminase, an enzyme found in white blood cells.

Donald Kohn, a microbiology, immunology and molecular genetics professor, and his team of researchers conducted a clinical trial on 10 children with bubble baby disease between 2009 and 2012 to develop the treatment, according to a study published this month.

The most common treatment of bubble baby disease is a twice-a-week injection of adenosine deaminase, according to a UCLA press release. It is a costly process that often cannot ensure the optimal functioning of the immune system.

In the trial, researchers extracted blood stem cells from each child’s bone marrow and modified these cells in the lab by inserting the gene responsible for producing adenosine deaminase. They later transplanted the corrected blood stem cells of each child into their bodies.

Nine of the children were cured, with the exception of one patient who was a teenager when receiving treatment. The cured children live normal lives with healthy immune systems that prevent common diseases such as the cold, according to the study.

According to the UCLA press release, the researchers’ next step is to seek approval from the Food and Drug Administration for the treatment.

Researchers at Kohn’s lab are currently adopting similar therapies to treat sickle cell disease and X-linked chronic granulomatous disease.

“My hope is that our current clinical trial for sickle cell disease, as well as the preclinical sickle cell research my lab continues to work on, will coalesce into a treatment that greatly improves, or even reverses the disease,” Kohn said in a press release.

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