Compositions and methods of treating facioscapulohumeral muscular dystrophy
US-2024400707-A1 · Dec 5, 2024 · US
US10967070B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10967070-B2 |
| Application number | US-201615056777-A |
| Country | US |
| Kind code | B2 |
| Filing date | Feb 29, 2016 |
| Priority date | Aug 29, 2013 |
| Publication date | Apr 6, 2021 |
| Grant date | Apr 6, 2021 |
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Provided herein are cell penetrating conjugates. The conjugates include a non-cell penetrating protein attached to a phosphorothioate nucleic acid or phosphorothioate polymer backbone, wherein the phosphorothioate nucleic acid or phosphorothioate polymer backbone enhances intracellular delivery of the non-cell penetrating protein. Also provided are compositions and kits comprising the cojugates.
Opening claim text (preview).
What is claimed is: 1. An antibody covalently attached to one or more phosphorothioate nucleic acids, wherein the one or more phosphorothioate nucleic acids are independently from 10 to 30 residues in length, wherein the antibody binds an intracellular target and wherein the antibody is non-cell penetrating in the absence of said one or more phosphorothioate nucleic acids. 2. The antibody of claim 1 , wherein each phosphorothioate nucleic acid is independently attached to a lysine, arginine, cysteine, or histidine of the antibody. 3. The antibody of claim 1 , wherein the intracellular target is a target of a disease selected from the group consisting of autoimmune disease, inflammatory disease, metabolic disorder, developmental disorder, cardiovascular disease, liver disease, intestinal disease, infectious disease, endocrine disease, neurological disorder, and cancer. 4. The antibody of claim 1 , wherein the intracellular target is a cancer target. 5. The antibody of claim 1 , wherein the intracellular target is selected from the group consisting of STAT3, exportin 7, and Src. 6. A cell comprising the antibody of claim 1 . 7. A pharmaceutical composition comprising the antibody of claim 1 and a pharmaceutically acceptable carrier. 8. A kit comprising the antibody of claim 1 or the pharmaceutical composition of claim 7 and instructions for use.
the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense · CPC title
the antibody targeting a material from animals or humans · CPC title
Protein; Peptide · CPC title
Sugars, nucleosides, nucleotides or nucleic acids · CPC title
DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology, C07H21/00); {Non-coding nucleic acids having a biological activity} · CPC title
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