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. An interfering RNA molecule-ligand conjugate comprising the sequence of SEQ ID NO: 4, wherein the ligand can bind to a transferrin receptor (TfR).
2. The interfering RNA molecule-ligand conjugate of claim 1 , wherein the ligand comprises a TfR binding domain of transferrin.
3. The interfering RNA molecule-ligand conjugate of claim 2 , wherein the ligand comprises the Tf C-lobe or a TfR-binding portion thereof.
4. The interfering RNA molecule-ligand conjugate of claim 1 , wherein the ligand comprises a TfR-binding peptide, a TfR-binding aptamer, or a TfR-specific antibody or a fragment thereof.
5. The interfering RNA molecule-ligand conjugate of claim 1 , wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.
6. A pharmaceutical composition comprising the interfering RNA molecule-ligand conjugate of claim 1 .