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NOTCH3 CADASIL Variant Receptor Aggregation Requires NOTCH3 Wild-Type Receptors: Identification of Highly Selective Inhibitors That Block the Process - PubMed

a day ago
  • #Therapeutic Inhibitors
  • #NOTCH3
  • #CADASIL
  • CADASIL is a monogenic autosomal dominant small vessel disease caused by mutations in NOTCH3 receptor.
  • Pathogenic NOTCH3 CADASIL variants require wild-type NOTCH3 receptors to form high molecular mass assemblies.
  • CADASIL mutations in EGFr 4 of NOTCH3 enhance JAGGED1 (JAG1)-dependent NOTCH3 transactivation.
  • Novel NOTCH3 inhibitors can block receptor aggregation and mutant NOTCH3 signaling, offering potential therapy.