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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

A Versatile Reporter Platform for Evaluating HDR- and NHEJ-Based Genome Editing in Airway Epithelial Cell Cultures Using an rAAV Vector

Friday, June 27, 2025
Soo Yeun Park
Therapeutic gene editing strategies utilize endogenous DNA repair pathways-nonhomologous end joining (NHEJ) or homology-directed repair (HDR)-to introduce targeted genomic modifications. Because HDR is restricted to dividing cells, whereas NHEJ functions in both dividing and non-dividing cells, NHEJ-based approaches are better suited for in vivo gene editing in the largely post-mitotic airway epithelium. Homology-independent targeted insertion (HITI), an NHEJ-based method, offers a promising...

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