IRBM cited in UniQure publication on using HTT-lowering therapies to slow down neurodegeneration in Huntington’s Disease
UniQure has just published a study in Science Translational Medicine using a Huntingtin (HTT)-lowering therapy.…
One of our studies featured in this publication, deals with an exploratory program aimed at identifying selective inhibitors of isoform-2 acid-sensing ion channels (ASIC-2). ASIC is a family of proton-activated ion channels widely expressed both in central and peripheral nervous system, as well as in cancer cells, and may represent a relevant target for drug discovery. Diminazene, a known ASIC inhibitor endowed with poor target specificity and negligible blood-brain barrier penetration, was used as a chemical starting point for optimization. Compound 2u was identified as the first selective and brain penetrant ASIC-2 inhibitor, as a useful tool to validate the role of ASIC-2 isoform in different type of neurodegenerative pathologies.
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