novel small molecules targeting hnRNP A1

Novel small molecules targeting heterogeneous nuclear ribonucleoprotein A1 (hnRNP A1) are being developed by the University of Saskatchewan and the University of British Columbia for the treatment of neurodegeneration in progressive multiple sclerosis (MS). Under homeostatic conditions, hnRNP A1 resides in the nucleus where it regulates RNA metabolism and splicing. In MS, hnRNP A1 becomes dysfunctional, mislocalizing to the cytoplasm and forming toxic aggregates, which drives neurite loss and neurodegeneration. These peptidomimetic small molecules are designed to bind hnRNP A1, correct its nucleocytoplasmic mislocalization, and inhibit aggregation, thereby preserving neuronal integrity and preventing disability progression. Preclinical studies in experimental autoimmune encephalomyelitis (EAE) models have demonstrated that these orally bioavailable compounds exhibit favorable brain penetration, lack of toxicity, and significant efficacy in reducing neurodegeneration.

Other names

hnRNP A1 nucleocytoplasmic mislocalization inhibitorshnRNP A1-specific small moleculespeptidomimetic small molecules targeting hnRNP A1