RNA interference (RNAi) is a potent and specific mechanism for regulating gene expression.
Harnessing RNAi to silence genes involved in disease holds promise for the development …

Purpose Infantile Pompe disease resulting from a deficiency of lysosomal acid α-glucosidase
(GAA) requires enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA). …

Mir-290 through mir-295 (mir-290–295) is a mammalian-specific microRNA (miRNA) cluster
that, in mice, is expressed specifically in early embryos and embryonic germ cells. Here, we …

Rhizomelic chondrodysplasia punctata (RCP) is a rare autosomal recessive disorder with
many associated medical complications. Prior to this study, natural history information about …

In the murine model of infection, a Mycobacterium tuberculosis mce1 operon mutant elicits an
aberrant granulomatous response, resulting in uncontrolled replication and failure to enter …

The high stress environment of law enforcement places officers at risk for a variety of mental
and physical health problems; however, officers are reluctant to seek out treatment. The …

βIV spectrin links ankyrinG (AnkG) and clustered ion channels at axon initial segments (AISs)
and nodes of Ranvier to the axonal cytoskeleton. Here, we report bi-allelic pathogenic …

Freehand 3D ultrasound allows intra-operative imaging of volumes of interest in a fast and
flexible way. However, the ultrasound device must be calibrated before it can be registered …

Mycobacterium tuberculosis causes a variety of clinical outcomes determined by host as
well as bacterial factors. M. tuberculosis disrupted in the mce1 operon causes increased …

A reliability generalization (RG) study was conducted on the Coopersmith Self-Esteem
Inventory (CSEI) to (a) examine the variability of reliability estimates across studies and (b) …