Publications by authors named "J Bradner"
Article Synopsis
- - Sickle cell disease (SCD) is a serious inherited condition caused by a mutation in the β-hemoglobin gene, and increasing fetal hemoglobin (HbF) levels can help reduce complications.
- - Researchers discovered two small molecules, dWIZ-1 and dWIZ-2, that act as molecular glue degraders to induce HbF by targeting a previously unrecognized repressor, the WIZ transcription factor.
- - These compounds effectively triggered HbF production in animal models, suggesting that targeting WIZ for degradation offers a promising and accessible new treatment approach for SCD.
Dopaminergic neurons of the substantia nigra exist in a persistent state of vulnerability resulting from high baseline oxidative stress, high-energy demand, and broad unmyelinated axonal arborisations. Impairments in the storage of dopamine compound this stress because of cytosolic reactions that transform the vital neurotransmitter into an endogenous neurotoxicant, and this toxicity is thought to contribute to the dopamine neuron degeneration that occurs Parkinson's disease. We have previously identified synaptic vesicle glycoprotein 2C (SV2C) as a modifier of vesicular dopamine function, demonstrating that genetic ablation of SV2C in mice results in decreased dopamine content and evoked dopamine release in the striatum.
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