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.…
In a recent publication for The Journal of Clinical Investigation, IRBM and our international collaborators report exciting findings regarding a potential treatment for Huntington’s disease.
We found elevated levels of the active proteinase ADAM10 in the presence of the polyglutamine-expanded huntingtin protein in both in vivo and in vitro Huntington’s disease models. An accumulation of overactive ADAM10 leads to excessive cleavage of the adhesion molecule N-CAD and loss of synaptic structure and connectivity, possibly mediating the cognitive dysfunction in patients with the disease. Our data indicates that inhibiting ADAM10 activity can be neuroprotective in Huntington’s disease treatment, restoring in vitro synaptic function and in vivo cognitive deficits. This may be a powerful treatment strategy to protect patients from the progressive loss of neurotransmission and ultimately cognitive decline.
The full article can be read here.