Primary Ciliary Dyskinesia Patient-Specific hiPSC-Derived Airway Epithelium in Air-Liquid Interface Culture Recapitulates Disease Specific Phenotypes In Vitro
Round 1
Reviewer 1 Report
very good manscript
Reviewer 2 Report
This is a very well-conceived study and the results have been presented systematically. I do not have any issues and I think the manuscript can be accepted for publication.
Reviewer 3 Report
The authors describe the establishment of a hiPSC-based primary ciliary dyskinesia in vitro disease model using patient lines containing mutations in the most frequently mutated and well described PCD-associated gene DNAH5 (41, 42), and in the recently as PCD-associated described gene NME5, respectively and validate the model for analizing the disease phenotype on a molecular, structural and functional level.
The model provides advantages in relation to previous describes models like non-aiway immortalized cell lines, differentiation protocols for generation of airway epithelium from hiPSCs including an intermediate 3D culture step, or airway-on-a-chip-technology. The author’s model platform utilizes a simple air-liquid interface culture system and is useful for throughput screening applications.
The manuscript is well written, and the authors describe very correctly the methodology used and the results obtained. The discussion is also adequate.