Defects in the PKD1 and PKD2 genes cause autosomal dominant polycystic kidney disease
(PKD) in~ 1 in 1000 adults worldwide. These genes encode polycystin-1 and polycystin-2, …

Eukaryotic cilia are assembled via intraflagellar transport (IFT) in which large protein particles
are motored along ciliary microtubules. The IFT particles are composed of at least 17 …

Inefficient knock-in of transgene cargos limits the potential of cell-based medicines. In this
study, we used a CRISPR nuclease that targets a site within an exon of an essential gene and …

Primary cilia project from the surface of most vertebrate cells and are thought to be sensory
organelles. Defects in primary cilia lead to cystic kidney disease, although the ciliary …

Intraflagellar transport (IFT), which is the bidirectional movement of particles within flagella,
is required for flagellar assembly. IFT particles are composed of ∼16 proteins, which are …

The intraflagellar transport (IFT) system is required for building primary cilia, sensory organelles
that cells use to respond to their environment. IFT particles are composed of about 20 …

Eukaryotic cells often use proteins localized to the ciliary membrane to monitor the
extracellular environment. The mechanism by which proteins are sorted, specifically to this …

Sensory functions of primary cilia rely on ciliary-localized membrane proteins, but little is known
about how these receptors are targeted to the cilium. To further our understanding of this …

The primary cilium plays a key role in the development of mammals and in the maintenance
of health. Primary cilia are assembled and maintained by the process of intraflagellar …

Odontochondrodysplasia (ODCD) is an unresolved genetic disorder of skeletal and dental
development. Here, we show that ODCD is caused by hypomorphic TRIP11 mutations, and …