Neeraj Sharma

The paper integrates clinical and primary cell data to show the CFTR function-phenotype relationship is logarithmic, not linear, with greater phenotypic gains at lower baseline function.

This study measures CFTR function in nasal airway cells from CF heterozygotes and controls, finding reduced CFTR activity in heterozygotes regardless of symptoms.

This study corrects a CFTR splice site variant in airway cells using base editors and nanoparticles, showing increased CFTR expression and functional rescue in multiple cell types.