Molecules for targeting compounds to various selected organs or tissues
The invention provides conjugates, comprising an organ, tissue or tumor cell homing molecule linked to a moiety. Such a moiety can be, for example, an oligonucleotide, small interfering RNA, gene, virus, protein, pharmaceutical or detectable agent. In addition the invention provides methods to diagnose or treat a pathology of the muscle or heart, by administrating to a subject having or suspected of having a pathology a molecule or conjugate that homes to, binds to and is taken up by the muscle cells or heart cells.
1. A conjugate of a peptide or peptidomimetic comprising the sequence or consisting of the sequence LGAQSNF (SEQ ID NO: 100) linked to a moiety selected from a biologically active moiety and a diagnostic moiety.
2. The conjugate according to claim 1 , wherein the biologically active moiety is selected from the group consisting of DNA, RNA or analogues thereof.
3. The conjugate according to claim 1 wherein the conjugate is a fusion protein comprising a peptide of SEQ ID NO: 100 and a therapeutically active protein and/or a diagnostic protein.
4. The conjugate according to claim 3 which further comprises a nuclear localisation signal.
5. A pharmaceutical composition comprising a conjugate according to claim 1 , in combination with a pharmaceutically acceptable carrier.
6. The pharmaceutical composition according to claim 5 wherein the pharmaceutical composition is for the treatment of a muscle-cell associated disorder including cardiac disorders.
7. The pharmaceutical composition according to claim 6 wherein the pharmaceutical composition is for the treatment of a myopathy, muscular dystrophy or muscle wasting disease.
8. The pharmaceutical composition according to claim 5 wherein the pharmaceutical composition is for the treatment of diabetes mellitus type II or obesity.
9. A molecule comprising a peptide or peptidomimetic comprising the sequence or consisting of the sequence LGAQSNF (SEQ ID NO: 100) and a linking portion which is not a peptide for linking the molecule to a biologically active moiety or a diagnostic moiety.
10. The conjugate according to claim 2 , wherein the biologically active molecule is a DNA or RNA analogue selected from the group consisting of 2′-O-alkyl, 2′-O -mehoxyethyl- , 2′-O-methyl, 2′-O-alkenyl (allyl), 2′-O-alkynyl nucleotides, locked nucleic acids (LNAs), peptide nucleic acids (PNAs), ethylene bridged nucleic acids (ENAs), phosphorothioate modified nucleotides, morpholino based nucleotides and combinations thereof.