Methods and compositions for treatment of myotonic dystrophy

摘要

In certain embodiments, the present invention provides compositions and methods for treating myotonic dystrophy.

主权项

1. A chimeric polypeptide comprising: (i) a functional fragment of an MBNL1 polypeptide, wherein the MBNL1 polypeptide comprises an amino acid sequence that is at least 95% identical to SEQ ID NO: 3, and wherein the functional fragment comprises amino acids 1-248 of the MBNL1 polypeptide, and (ii) a targeting moiety, wherein the targeting moiety comprises an antibody or antigen-binding fragment, wherein the targeting moiety transits cellular membranes via an equilibrative nucleoside transporter 2 (ENT2) transporter; and wherein the chimeric polypeptide is capable of binding CUG repeats.