Developing new gene therapy for lung surfactant deficiency
Professor Deborah Gill of the University of Oxford is carrying out laboratory research that could ultimately lead to a vital new treatment for newborn babies with lung surfactant deficiency.
Lung surfactant deficiency is a rare condition that causes breathing problems shortly after birth. It's caused by faults in a gene called ABCA3, which is responsible for the production of a surfactant, a compound that lines the lung tissue. Without surfactant, the lungs collapse, and the baby cannot breathe without mechanical ventilation.
With no cure and few treatments, new life-saving therapies are desperately needed to provide hope for children and their families affected by this devastating disease.
"Our goal is to develop a new gene therapy for babies with severe breathing problems caused by faults in the ABCA3 gene."
Professor Gill and her team will work in the laboratory to test the effectiveness of using a harmless virus to deliver a working copy of the ABCA3 gene into the baby's lungs.
The researchers hope that this work will show that replacing the faulty gene will enable babies to make their own lung surfactant, which should help them to breathe independently without the need for ventilation.
If the results prove promising, the next step would be a clinical trial to test the safety and effectiveness of delivering the treatment directly into babies’ lungs via the ventilator breathing tubes.
If successful, this gene therapy could transform the outlook for babies born with severe lung surfactant deficiency. It could also provide benefits for children with milder symptoms of the disease.
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