Tracheal

Research Spotlight

Dr. John F. Engelhardt research focuses on the molecular basis of CF and the development of gene therapies for the disease. Notably, Dr. Engelhardt's laboratory was the first to clone ferrets, creating new animal models that have enhanced the study of CF pathologies and potential treatments.

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Research Papers

Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRDR gene delivery to adult ferret lungs by Yinghua Tang - Anatomy and Cell Biolgy

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Pilot Programs

Pilot programs  provides seed money for research that has not yet reached the stage of maturity necessary for funding by traditional NIH mechanisms such as RO1s and PO1s. Such research ideas should be considered innovative and may potentially be high risk.

Latest Publications

Lentiviral-mediated gene complementation rescues pathogenic <em>ABCA3</em> variants

Friday, September 5, 2025
Ashley L Cooney
The ATP-binding cassette subfamily A member 3 (ABCA3) protein on the limiting membrane of lamellar bodies in alveolar type 2 (AT2) cells transports phospholipids required for pulmonary surfactant assembly. ABCA3 deficiency results from biallelic pathogenic variants in ABCA3 and causes progressive neonatal respiratory failure or childhood interstitial lung disease (chILD). Supportive/compassionate care or lung transplantation are the only current definitive treatments for ABCA3 deficiency and...

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