Back to results
US 9,114,178 B2Granted
Methods and compositions for treatment of myotonic dystrophy
AssigneeValerion Therapeutics, LLC
InventorDustin D. Armstrong
Life of this patent
Estimating the term…
Abstract
In certain embodiments, the present invention provides compositions and methods for treating myotonic dystrophy.
Claims56
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.
A nucleic acid construct, comprising a nucleotide sequence that encodes 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, wherein the functional fragment comprises amino acids 1-248 of the MBNL1 polypeptide, and wherein the functional fragment is operably linked to a nucleotide sequence that encodes 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;
- wherein the nucleic acid construct encodes a chimeric polypeptide having MBNL biological activity and having the targeting activity of the targeting moiety;
- and wherein the chimeric polypeptide is capable of binding CUG repeats.
A method of delivering a chimeric polypeptide into a cell, comprising contacting a cell with a chimeric polypeptide, which chimeric polypeptide comprises:
- (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.
A chimeric polypeptide comprising:
- (i) a functional fragment of an MBNL1 polypeptide, wherein the functional fragment comprises amino acids 1-248 of a human 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.
Dependencies are inferred from the claim text.