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The team aim to develop a new gene therapy for babies born with lung surfactant deficiency, a rare condition causing them to struggle to breathe. Without a lung transplant, less than 1 in 5 children will live to 5 years. Research could lead to a brighter future for children and their families.
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Beneficiaries
Situation
Lung surfactant is a complex mixture of fats and proteins that lines the lung tissue and makes breathing easy. Without this, the lungs can collapse, leading to life-threatening breathing difficulties. Research has identified faults in several genes that can this, including ABCA3, which provides the instructions for making a protein involved in surfactant production. Faults in this gene can cause life-threatening breathing difficulties in newborn babies or milder symptoms in older children.
Solution
The researchers plan to deliver a working copy of the ABCA3 gene into the baby’s lung cells. By enabling babies to make normal lung surfactant, this treatment should help them to breathe without mechanical ventilation. The next step would be a clinical trial to test the safety and effectiveness of delivering the treatment directly through the ventilator breathing tube. If successful, this gene therapy could transform the outlook for babies born with severe lung surfactant deficiency.