Mutations in the GNAO1 gene are tied to neurological disorders characterized by movement abnormalities and developmental delay. GNAO1 encodes the protein guanine nucleotide-binding protein G(o) subunit α, which is highly expressed in the brain. Among the mutations, R209H results in dystonia, choreoathetosis and developmental delay without seizures.

Aortic aneurysm is characterized by enlargement of the aorta, as well as vascular inflammation and damage to the extracellular matrix.

Researchers from Washington University School of Medicine performed studies to assess the role of ZBTB46, a repressive transcription factor and a marker for classical dendritic cells (DCs), in tumor angiogenesis.

Researchers from Excision Biotherapeutics Inc. and affiliated organizations have reported the development of a novel CRISPR/Cas9-mediated genome editing therapy – EBT-104 – for the treatment of latent herpes simplex virus 1 (HSV-1) keratitis.

Find Therapeutics Inc. has obtained FDA clearance of its IND application for FTX-101, a first-in-class remyelinating agent that aims to restore vision in people with chronic optic neuropathy.

Glucocorticoids are the standard treatment to treat inflammation in asthma, however, they are often associated with cardiovascular disease, osteoporosis or immune suppression risk with chronic use. Novel strategies to combine with traditional treatments, such as those intended to restore hydrogen sulfide (H2S) levels, have emerged as options for treating severe uncontrolled asthma with frequent exacerbations.

Restoring glucose metabolism in astrocytes, which is impaired in Alzheimer’s disease (AD), has a direct effect on neurons, which replenish their fuel supply and resume synaptic activity. A group of scientists from Stanford University School of Medicine has revealed the pathway that explains where this efflux is interrupted and which molecules restore it in mouse models with amyloid and tau pathology. Their findings could help prevent the progression of this neurodegenerative disease.