By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures. The channels are expressed when the promoter is turned on by excessive neuronal activity, and so “we can’t stop the first seizures,” Dimitri Kullmann told BioWorld.

Charcot-Marie-Tooth disease (CMT) is a heterogeneous group of congenital disorders characterized by peripheral neuropathy.

Researchers in China have discovered a new therapeutic target, and identified a rapid-acting lead compound that may overcome the drawbacks of current antidepressants. The research team, based at Nanjing Medical University’s School of Pharmacy, designed a fast-acting antidepressant that works by disrupting the interaction between the serotonin transporter (SERT) and neuronal nitric oxide synthase (nNOS) in the dorsal raphe nucleus. The dorsal raphe nucleus (DRN) of the midbrain influences a number of central nervous system processes, and the main transmitter of the DRN is serotonin, the primary target for most antidepressants. Neurons communicate with each other by releasing a series of neurotransmitters into the synaptic space, and these transmitted electrical signals ultimately determine feelings, thoughts and actions, explained co-lead study author Qi-Gang Zhou. The study was published in Science on Oct. 28, 2022.

Dystroglycanopathies are a subset of rare congenital muscular dystrophies (CMDs) caused by dysregulation in the assemblage of glycans on the α-dystroglycan (α-DG) transmembrane glycoprotein.