Neutrophil elastase: Nonsense lost in translation.

Horwitz, Marshall S · Cell Stem Cell · 2021

editorial · Level V

Where this comes from

Abstract

In this issue of Cell Stem Cell, Daniel Bauer and colleagues investigate the pathogenesis of ELANE-associated severe congenital neutropenia (SCN) and describe two potentially universal gene correction strategies for autosomal dominant disorders (Rao et al., 2021). One exploits nonsense-mediated decay to prevent translation of the mutant allele. The other unexpectedly blocks translation by shortening the 3'-UTR.

Medical subject headings