Stem cell transplant could help cystic fibrosis patients

September 17, 2018

Healthy airway stem cells could be used one day to replace the damaged cells that cause cystic fibrosis, providing a new form of treatment for the genetic disorder.

Researchers from the University of Adelaide used techniques similar to those employed for stem cell treatments of autoimmune conditions to make the discovery, according to a report on their study published in Stem Cell Research & Therapy and summarized at Rare Disease Report.

In cystic fibrosis, patients’ lungs and digestive systems become blocked by an overproduction of mucus. There is no cure for cystic fibrosis.

In the study, the Australian researchers collected adult stem cells from the lungs of adult cystic fibrosis patients, then corrected the gene using gene therapy. They tested the theory in mice.

The airway stem cells successfully transplanted in just two hours, much more quickly than the researchers anticipated.

“The premise of this mode of cystic fibrosis gene therapy is that it would be able to prevent the onset of lung disease in young patients or halt disease progression in older patients with existing disease,” the study’s authors wrote in their conclusion.

The GenCure BioManufacturing Center works with scientists doing research into stem cell therapies for a range of conditions, from preclinical studies to commercialization.

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