Spinal muscular atrophy (SMA) is a severe neuromuscular disorder due to a defect in the
survival motor neuron 1 (SMN1) gene. Its incidence is approximately 1 in 11,000 live births. In …

Whether the brain tumor medulloblastoma originates from stem cells or restricted progenitor
cells is unclear. To investigate this, we activated oncogenic Hedgehog (Hh) signaling in …

Ceramides contribute to the lipotoxicity that underlies diabetes, hepatic steatosis, and heart
disease. By genetically engineering mice, we deleted the enzyme dihydroceramide …

Progression of diabetic nephropathy to end-stage kidney disease is mediated by a host of
pro-cesses, but none is as important as the gradual, inexorable scarring of the renal …

Mechanisms of brain and retinal iron homeostasis have become subjects of increased interest
after the discovery of elevated iron levels in brains of patients with Alzheimer's disease …

This is the second half of a two-part document updating the standard of care
recommendations for spinal muscular atrophy published in 2007. This part includes updated …

Background The objective of this study is to conduct a systematic review of studies reporting
the frequency of neurocysticercosis (NCC) worldwide. Methods/Principal Findings PubMed, …

Despite increased use of human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs)
for drug development and disease modeling studies, methods to generate large, …

Glutamatergic and GABAergic neurons mediate much of the excitatory and inhibitory
neurotransmission, respectively, in the vertebrate nervous system. The process by which …

Objectives To characterize the prevalence of plasmid-mediated quinolone resistance
determinants qnr and aac(6′)-Ib-cr in extended-spectrum β-lactamase (ESBL)-producing …