Knottins: Disulfide-bonded therapeutic and diagnostic peptides

Drug Discovery Today: Technologies

Volumn 9, Issue 1, 2012, Pages e3-

  Moore, Sarah J ^a   Leung, Cheuk Lun ^a   Cochran, Jennifer R ^a  

^a STANFORD CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGOUTI RELATED PROTEIN; ANKYRIN; AVIMER DERIVATIVE; CALCIUM CHANNEL; CONANTOKIN G; CYCLOTIDE; DIAGNOSTIC AGENT; EPITOPE; EPTIFIBATIDE; FIBRONECTIN; FLUORODEOXYGLUCOSE F 18; KNOTTIN; LIPOCALIN; OMEGA CONOTOXIN MVIIA; PEGDINETANIB; PEPTIDE DERIVATIVE; RECOMBINANT THROMBOPOIETIN; TRYPSIN INHIBITOR; UNCLASSIFIED DRUG; VASCULOTROPIN; VENOM;

ANTIMICROBIAL ACTIVITY; ARTICLE; AUTOIMMUNE DISEASE; BIOLOGICAL ACTIVITY; BRAIN ISCHEMIA; CACHEXIA; CANCER THERAPY; CHRONIC PAIN; CROHN DISEASE; CYSTINE KNOT MOTIF; DIABETIC MACULAR EDEMA; DISULFIDE BOND; DRUG DISTRIBUTION; DRUG HALF LIFE; DRUG RECEPTOR BINDING; DRUG RESEARCH; DRUG STABILITY; DRUG STRUCTURE; DRUG TUMOR LEVEL; GLIOBLASTOMA; HUMAN; IMMUNOGENICITY; MOLECULAR IMAGING; NERVE CELL LESION; NEUROPROTECTION; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); POSITRON EMISSION TOMOGRAPHY; PROTEIN DEGRADATION; PROTEIN ENGINEERING; RETINA MACULA AGE RELATED DEGENERATION; THERMOSTABILITY; THROMBOCYTE AGGREGATION;

EID: 84865689360     PISSN: None     EISSN: 17406749     Source Type: Journal    
DOI: 10.1016/j.ddtec.2011.07.003     Document Type: Review

References (57)

1. Craik, D.J. et al. (2007) Potential therapeutic applications of the cyclotides and related cystine knot mini-proteins. Expert Opin. Invest. Drugs 16, 595-604 (Pubitemid 46723268)
2. Kolmar, H. (2009) Biological diversity and therapeutic potential of natural and engineered cystine knot miniproteins. Curr. Opin. Pharmacol. 9, 608-614
3. Kolmar, H. (2010) Engineered cystine-knot miniproteins for diagnostic applications. Expert Rev. Mol. Diagn. 10, 361-368
4. Kolmar, H. (2008) Alternative binding proteins: Biological activity and therapeutic potential of cystine-knot miniproteins. FEBS J. 275, 2684-2690
5. Gracy, J. et al. (2008) KNOTTIN: The knottin or inhibitor cystine knot scaffold in 2007. Nucleic Acids Res. 36, D314-D319 (Pubitemid 351175212)
6. Williams, J.A. et al. (2008) Ziconotide: An update and review. Expert Opin. Pharmacother. 9, 1575-1583 (Pubitemid 352005917)
7. Ollmann, M.M. et al. (1997) Antagonism of central melanocortin receptors in vitro and in vivo by agouti-related protein. Science 278, 135-138 (Pubitemid 27446294)
8. Joppa, M.A. et al. (2007) Central infusion of the melanocortin receptor antagonist agouti-related peptide (AgRP(83-132)) prevents cachexiarelated symptoms induced by radiation and colon-26 tumors in mice. Peptides 28, 636-642 (Pubitemid 46205015)
9. Pysz, M.A. et al. (2010) Molecular imaging: Current status and emerging strategies. Clin. Radiol. 65, 500-516
10. Veiseh, M. et al. (2007) Tumor paint: A chlorotoxin:Cy5.5 bioconjugate for intraoperative visualization of cancer foci. Cancer Res. 67, 6882-6888 (Pubitemid 47105535)
11. Christmann, A. et al. (1999) The cystine knot of a squash-type protease inhibitor as a structural scaffold for Escherichia coli cell surface display of conformationally constrained peptides. Protein Eng. 12, 797-806 (Pubitemid 29472087)
12. Reiss, S. et al. (2006) Inhibition of platelet aggregation by grafting RGD and KGD sequences on the structural scaffold of small disulfide-rich proteins. Platelets 17, 153-157 (Pubitemid 43791626)
13. Kimura, R.H. et al. (2009) Engineered cystine knot peptides that bind alphav beta3, alphav beta5, and alpha5 beta1 integrins with lownanomolar affinity. Proteins 77, 359-369
14. Lahti, J.L. et al. (2009) Interrogating and predicting tolerated sequence diversity in protein folds: Application to E elaterium trypsin inhibitor-II cystine-knot miniprotein. PLoS Comput. Biol. 5, e1000499
15. Silverman, A.P. et al. (2011) Cystine-knot peptides engineered with specificities for alphaIIb beta3 or alphaIIb beta3 and alphav beta3 integrins are potent inhibitors of platelet aggregation. J. Mol. Recognit. 24, 127-135
16. Silverman, A.P. et al. (2009) Engineered cystine-knot peptides that bind alphav beta3 integrin with antibody-like affinities. J. Mol. Biol. 385, 1064
17. Krause, S. et al. (2007) Grafting of thrombopoietin-mimetic peptides into cystine knot miniproteins yields high-affinity thrombopoietin antagonists and agonists. FEBS J. 274, 86-95 (Pubitemid 44952900)
18. Werle, M. et al. (2006) The potential of cystine-knot microproteins as novel pharmacophoric scaffolds in oral peptide drug delivery. J. Drug Target. 14, 137-146 (Pubitemid 43878822)
19. Kimura, R.H. et al. (2009) Engineered knottin peptides: A new class of agents for imaging integrin expression in living subjects. Cancer Res. 69, 2435-2442.
20. Jiang, L. et al. (2010) Evaluation of a 64Cu-labeled cystine-knot peptide based on agouti-related protein for PET of tumors expressing alphavbeta3 integrin. J. Nucl. Med. 51, 251-258
21. Miao, Z. et al. (2009) An engineered knottin peptide labeled with 18F for PET imaging of integrin expression. Bioconjug. Chem. 20, 2342-2347
22. Nielsen, C.H. et al. (2010) PET imaging of tumor neovascularization in a transgenic mouse model with a novel 64Cu-DOTA-knottin peptide. Cancer Res. 70, 9022-9030
23. Miao, Z. et al. (2010) Protein scaffold-based molecular probes for cancer molecular imaging. Amino Acids 10.1007/s00726-010-0503-9
24. Kimura, R.H. et al. (2010) A dual-labeled knottin peptide for PET and nearinfrared fluorescence imaging of integrin expression in living subjects. Bioconjug. Chem. 21, 436-444
25. Willmann, J.K. et al. (2010) Targeted contrast-enhanced ultrasound imaging of tumor angiogenesis with contrast microbubbles conjugated to integrin-binding knottin peptides. J. Nucl. Med. 51, 433-440
26. Jiang, L. et al. (2010) Preliminary evaluation of 177Lu-labeled knottin peptides for integrin receptor-targeted radionuclide therapy. Eur. J. Nucl. Med. Mol. Imaging 38, 613-622
27. Daly, N.L. and Craik, D.J. (2011) Bioactive cystine knot proteins. Curr. Opin. Chem. Biol. 15, 362-368
28. Wang, C.K. et al. (2008) CyBase: A database of cyclic protein sequences and structures, with applications in protein discovery and engineering. Nucleic Acids Res. 36, D206-D210 (Pubitemid 351149723)
29. Greenwood, K.P. et al. (2007) The cyclic cystine knot miniprotein MCoTIII is internalized into cells by macropinocytosis. Int. J. Biochem. Cell Biol. 2252-2264 (Pubitemid 47484280)
30. Clark, R.J. et al. (2006) Structural plasticity of the cyclic-cystine-knot framework: Implications for biological activity and drug design. Biochem. J. 85-93 (Pubitemid 43259658)
31. Gunasekera, S. et al. (2008) Engineering stabilized vascular endothelial growth factor-A antagonists: Synthesis, structural characterization, and bioactivity of grafted analogues of cyclotides. J. Med. Chem. 51, 7697-7704
32. Thongyoo, P. et al. (2009) Potent inhibitors of beta-tryptase and human leukocyte elastase based on the MCoTI-II scaffold. J. Med. Chem. 52, 6197-6200
33. Clark, R.J. et al. (2005) Engineering stable peptide toxins by means of backbone cyclization: Stabilization of the alpha-conotoxin MII. Proc. Natl. Acad. Sci. U.S.A. 102, 13767-13772 (Pubitemid 41377653)
34. Clark, R.J. et al. (2010) The engineering of an orally active conotoxin for the treatment of neuropathic pain. Angew. Chem. Int. Ed. Engl. 49, 6545-6548
35. Clark, R.J. et al. (2010) Cyclization of conotoxins to improve their biopharmaceutical properties. Toxicon 10.1016/j.toxicon.2010.12.003
36. Akcan, M. et al. (2011) Chemical re-engineering of chlorotoxin improves bioconjugation properties for tumor imaging and targeted therapy. J. Med. Chem. 54, 782-787
37. Colgrave, M.L. and Craik, D.J. (2004) Thermal, chemical, and enzymatic stability of the cyclotide kalata B1: The importance of the cyclic cystine knot. Biochemistry 43, 5965-5975 (Pubitemid 38669464)
38. Heitz, A. et al. (2008) Knottin cyclization: Impact on structure and dynamics. BMC Struct. Biol. 8, 54
39. Gebauer, M. and Skerra, A. (2009) Engineered protein scaffolds as nextgeneration antibody therapeutics. Curr. Opin. Chem. Biol. 13, 245-255
40. Wu, A.M. and Senter, P.D. (2005) Arming antibodies: Prospects and challenges for immunoconjugates. Nat. Biotechnol. 23, 1137-1146 (Pubitemid 41486395)
41. Thurber, G.M. et al. (2007) Theoretic criteria for antibody penetration into solid tumors and micrometastases. J. Nucl. Med. 48, 995-999 (Pubitemid 46909929)
42. Chames, P. et al. (2009) Therapeutic antibodies: Successes, limitations and hopes for the future. Br. J. Pharmacol. 157, 220-233
43. Justice, A. et al. [Neurex Corporation] (1999) Methods for producing analgesia. USP5859186
44. Bakker, A. et al. [Amgen Mountain View Inc.] (2010) IL-6 binding proteins. USP7786262
45. Silverman, J. et al. (2005) Multivalent avimer proteins evolved by exon shuffling of a family of human receptor domains. Nat. Biotechnol. 23, 1556-1561 (Pubitemid 41752934)
46. Löfblom, J. et al. (2010) Affibody molecules: Engineered proteins for therapeutic, diagnostic and biotechnological applications. FEBS Lett. 584, 2670-2680
47. Nilsson, F. et al. [Affibody AB] (2010) Polypeptides having binding affinity for Her2. US Patent application 20100048868
48. Bloom, L. and Calabro, V. (2009) FN3: A new protein scaffold reaches the clinic. Drug Discov. Today 14, 949-955
49. Yan, C. et al. [Bristol-Myers Squibb Company] (2010) Inhibitors of type 2 vascular endothelial growth factor receptors. USP7858739
50. Stumpp, M.T. et al. (2008) DARPins: A new generation of protein therapeutics. Drug Discov. Today 13, 695-701
51. Tobias, S.M. et al. [University of Zurich] (2008) Collection of repeat proteins comprising repeat modules. USP7417130
52. Skerra, A. (2008) Alternative binding proteins: Anticalins - Harnessing the structural plasticity of the lipocalin ligand pocket to engineer novel binding activities. FEBS J. 275, 2677-2683 (Pubitemid 351678679)
53. Skerra, A. et al. [Pieres AG] (2011) Muteins of human tear lipocalin. USP7893208B2
54. Schmoldt, H.U. et al. (2005) A fusion protein system for the recombinant production of short disulfide bond rich cystine knot peptides using barnase as a purification handle. Protein Expr. Purif. 39, 82-89
55. Tolmachev, V. (2008) Imaging of HER-2 overexpression in tumors for guiding therapy. Curr. Pharm. Des. 14, 2999-3019
56. Wu, A.M. et al. (2000) High-resolution microPET imaging of carcinoembryonic antigen-positive xenografts by using a copper-64- labeled engineered antibody fragment. Proc. Natl. Acad. Sci. U.S.A. 97, 8495-8500 (Pubitemid 30639701)
57. Zahnd, C. et al. (2010) Efficient tumor targeting with high-affinity designed ankyrin repeat proteins: Effects of affinity and molecular size. Cancer Res. 70, 1595-1605