Adiponectin (ADIPOQ) is an adipocyte-derived hormone that promotes insulin sensitivity, inhibits cell death and decreases inflammation. Previous studies identified common variants in the ADIPOQ gene that have been associated with aberrant adiponectin levels, obesity, type 2 diabetes and diabetic kidney disease. In a recent study, the first multigenerational family was identified as harboring a protein-truncating mutation in the ADIPOQ gene, p.Gly93GlufsTer73, that cosegregated with diabetes and end-stage renal disease.
Cold Spring Harbor Laboratory, Vichem Chemie Research and The Feinstein Institute for Medical Research have described dual-specificity tyrosine-(Y)-phosphorylation regulated kinase 1A (DYRK1A) inhibitors reported to be useful for the treatment of cancer, Down syndrome, mild cognitive impairment and Alzheimer's disease.
TFChem SAS has described new cyclic glycoaminoacid derivatives reported to be useful for the treatment of dry skin, atopic dermatitis, aging, inflammatory disorders, fibrosis and psoriasis, among other disorders.
Neuron-targeting of caveolin-1 (Cav-1) using AAV9 delivery and a synapsin-driven Cav-1 engineered construct, SynCav1, showed therapeutic potential in the hSOD1G93A model of familial amyotrophic lateral sclerosis (ALS).
Ryvu Therapeutics has outlined its plans over the next 2 years to advance one preclinical program into phase I trials, strengthen its synthetic lethality platform and accelerate its early pipeline. The company is currently leading multiple initiatives in the area of synthetic lethality.
In cancer, aberrant signaling pathways can evade therapy; activation of oncogenic PI3K and MAPK signaling has inspired researchers to develop molecularly targeted drugs, such as LP-182 (University of Michigan), a multitargeted kinase inhibitor that researchers have tested in vitro and in vivo for the treatment of myelofibrosis.
To develop a safe and effective respiratory syncytial virus (RSV) vaccine for use in early life, Boston Children's Hospital scientists and their collaborators developed CAF-08, a liposomal formulation consisting of combined TLR7 (3M-052) and Mincle (trehalose-6,6-dibehenate [TDB]) agonists and the RSV pre-F antigen.
Tumor necrosis factor receptor-associated factor 3 (TRAF3) is a cytoplasmic protein that controls signal transduction from different receptor families, and plays a key role in regulating immunity.
A fusion protein removed beta-amyloid plaque without producing the neurotoxic inflammation associated with other treatments, such as aducanumab immunotherapy. It is based on the alphaA Beta-Gas6 fusion protein developed in a mouse model of Alzheimer's disease (AD) by a team of researchers at The Korea Advanced Institute of Science and Technology (KAIST) in South Korea.