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Stem Cell Research Gene Therapy

Stem Cell Research Breakthrough For Cystic Fibrosis

6 months, 3 weeks ago

3633  0
Posted on Aug 04, 2018, 9 p.m.

University of Adelaide researchers have made a huge breakthrough in the fight against cystic fibrosis by successfully replacing cells causing the chronic illness with healthy ones using cell transplantation therapy typically used in bone marrow transplants treating immunodeficiency, as published in the journal Stem Cell Research and Therapy.

2,500 babies are born with cystic fibrosis in Australia alone and the defective gene causing the condition is carried by 1 in every 25 people, should both parents carry the gene chances of the child being born with the illness is 25% which causes mucus to build in the lungs and digestive system impairing breathing and increasing risk of chest infection. Currently there is no known cure for the chronic condition which affects worldwide an estimated 70,000 people who must undergo a range of treatments including physiotherapy to clear airways and use drugs to aid digestion.

This new study shows that transplanted stem cells pass on their healthy genes to daughter cells leading to the airways being replenished with healthy cells. The transplantation method involved using a marker gene in place of corrective cystic fibrosis gene being successful in mouse models via implanting harvested adult stem cells from lungs of CF patients and correcting them with gene therapy to then reintroduce back into the patient. Dr. Nigel Farrow says key to success was the innovative approach involving eliminating existing surface cells to create space for new healthy cells to be introduced. Should the technique be perfected it could accelerate research to improve lives of CF patients and potentially combat the chronic life limiting illness.

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