Rev-Erb-α and Rev-Erb-β are nuclear receptors that regulate the expression of genes involved
in the control of circadian rhythm 1 , 2 , metabolism 3 , 4 and inflammatory responses 5 . …

A significant current challenge in human genetics is the identification of interacting genetic
loci mediating complex polygenic disorders. One of the best characterized polygenic …

Activation of liver X receptors (LXRs) with synthetic agonists promotes reverse cholesterol
transport and protects against atherosclerosis in mouse models. Most synthetic LXR agonists …

Lafora disease is a fatal progressive myoclonus epilepsy. At root, it is due to constant
acquisition of branches that are too long in a subgroup of glycogen molecules, leading them to …

… Grossman then discussed the Ionis neurological drug pipeline that includes programs on
Huntington's disease, amyotrophic lateral sclerosis, and Alzheimer's disease. She presented …

Goblet cell metaplasia, excessive mucus production, and inadequate mucus clearance
accompany and exacerbate multiple chronic respiratory disorders, such as asthma and chronic …

Longer glucan chains tend to precipitate. Glycogen, by far the largest mammalian glucan
and the largest molecule in the cytosol with up to 55 000 glucoses, does not, due to a highly …

… Tamar Grossman, Director of Translational Medicine at Ionis Pharmaceuticals, and Dr.
Minassian updated the group on their efforts in using ASOs to downregulate glycogen synthase …

A family of genes encoding membrane proteins with a unique structure has been identified
in DNA and cDNA clones of various eukaryotes ranging from yeast to human. The nucleotide …

Systemic lupus erythematosus is an autoimmune disease that manifests in widespread
complement activation and deposition of complement fragments in the kidney. The complement …