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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

Theratyping as a tool to guide clinical decision-making in patients with suspected cystic fibrosis and variants currently ineligible for CFTR modulator therapy

Saturday, November 15, 2025
Ryan M Gannon
The combination of elexacaftor/tezacaftor/ivacaftor (ETI) is effective in people with cystic fibrosis (CF) with at least one F508del-CFTR allele; however, approximately 10% have variants without established ETI responsiveness and are ineligible for therapy. Additionally, diagnostic confirmation or exclusion of CF can be challenging in individuals with compatible symptoms, CFTR variants of unknown significance, and indeterminate sweat chloride or nasal potential difference (NPD) results. We...

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