Transferrin/transferrin receptor-mediated siRNA delivery
View Patent ↗The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a transferrin receptor (TfR). Therapeutic uses for the conjugates are also provided.
1. A composition comprising an interfering RNA molecule-ligand conjugate, wherein the ligand comprises a peptide comprising a transferrin receptor (TfR) binding domain of transferrin (Tf) that is covalently linked to a protamine, wherein the protamine is covalently linked to the interfering RNA, and wherein the interfering RNA is able to mediate RNA interference in a cell.
2. The composition of claim 1 , wherein the ligand comprises the full length TfR binding domain of transferrin.
3. The composition of claim 1 , wherein the ligand comprises the transferrin C-lobe.
4. The composition of claim 2 , wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.
5. The composition of claim 3 , wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.
6. The composition of claim 4 , wherein the interfering RNA molecule is a siRNA.
7. The composition of claim 5 , wherein the interfering RNA molecule is a siRNA.