ANTISENSE CONJUGATES FOR DECREASING EXPRESSION OF DMPK

摘要

The disclosure provides novel conjugates comprising antisense oligonucleotides that hybridize to a DMPK transcript and a 3E10 antibody or binding fragment thereof. Also considered are these conjugates further comprising MBNL1 polypeptides. Methods of treating myotonic dystrophy using these conjugates and kits comprising these conjugates are also considered. Wherein the conjugates are suitable for delivery to muscle cells.

主权项

1. A conjugate comprising
an antisense oligonucleotide that hybridizes to a DMPK transcript; and an antibody or antigen binding fragment selected from: monoclonal antibody 3E10, or a variant thereof that retains the cell penetrating activity of 3E10, or a variant thereof that binds the same epitope as 3E10, or an antibody that has substantially the same cell penetrating activity as 3E10 and binds the same epitope as 3E10, or an antigen binding fragment of any of the foregoing.