The present invention is directed to the discovery that the heterogeneous nuclear ribonucleprotein H (hnRNP H) is capable of binding mutant myotonic dystrophy (DM) protein kinase (DMPK) mRNA. The present invention is also directed to the discovery that modulation of the expression of hnRNP H results in reduced nuclear retention of the mutant DMPK mRNA. The present invention is further directed to screening compounds to identify drugs useful for treating DM type 1 (DM1).
Modulation of hnRNP H and treatment of DM1