Defining Functional Correction Thresholds in Primary Ciliary Dyskinesia for Effective Gene Therapies
Beck E. Fitzpatrick, Brett J. Wineinger, Jason E. Babcock, Erik J. Quiroz, Emily C. Liu, Lalit Kumar Gautam, Alejandro A. Pezzulo, Thomas O. Moninger, David Kyle Meyerholz, Douglas B. Hornick, Amy L. Ryan
This study defines a functional correction threshold for gene therapies in PCD by analyzing how mixed populations of mutant and wild-type ciliated cells affect mucociliary transport.
bioRxiv · Feb 20, 2026 · PreprintCell Biology