The therapeutic potential of modulators of the Hedgehog-Gli signaling pathway

Journal of Biology

Volumn 1, Issue 2, 2002, Pages

  Stecca, Barbara ^a   Ruiz I Altaba, Ariel ^a  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

SONIC HEDGEHOG PROTEIN; TRANSCRIPTION FACTOR GLI1; GLI PROTEIN; ONCOPROTEIN; TRANSACTIVATOR PROTEIN; TRANSCRIPTION FACTOR;

ENZYME ACTIVATION; ENZYME REPRESSION; HUMAN; MEMBRANE BINDING; NONHUMAN; ORGANOGENESIS; PRIORITY JOURNAL; PROTEIN ANALYSIS; PROTEIN BINDING; PROTEIN FUNCTION; REGULATORY MECHANISM; SHORT SURVEY; SIGNAL TRANSDUCTION; AGONISTS; ANIMAL; ANTAGONISTS AND INHIBITORS; DRUG EFFECTS; METABOLISM;

ERINACEIDAE;

ANIMALS; HEDGEHOG PROTEINS; HUMANS; ONCOGENE PROTEINS; SIGNAL TRANSDUCTION; TRANS-ACTIVATORS; TRANSCRIPTION FACTORS;

EID: 33749368571     PISSN: 14785854     EISSN: 14754924     Source Type: Journal    
DOI: 10.1186/1475-4924-1-9     Document Type: Short Survey

References (36)

1. Frank-Kamenetsky M, Zhang XM, Bottega S, Guicherit O, Wichterle H, Dudek H, Bumcrot D, Wang FY, Jones S, Shulok J, Rubin LL, Porter JA: Small molecule modulators of hedgehog signaling: identification and characterization of smoothened agonists and antagonists. J Biol 2002, 1:10.
2. Ho KS, Scott MP: Sonic hedgehog in the nervous system: functions, modifications and mechanisms. Curr Opin Neurobiol 2002, 12:57-63.
3. Nybakken K, Perrimon N: Hedgehog signal transduction: recent findings. Curr Opin Genet Dev 2002, 12:503-511.
4. Ruiz i Altaba A, Palma V, Dahmane N: Hedgehog-Gli signalling and the growth of the brain. Nat Rev Neurosci 2002, 3:24-33.
5. Mullor J, Sanchez P, Ruiz i Altaba A: Pathways and consequences: Hedgehog signaling in human diseases. Trends Cell Biol, in press.
6. Roessler E, Belloni E, Gaudenz K, Jay P, Berta P, Scherer SW, Tsui LC, Muenke M: Mutations in the human sonic hedgehog gene cause holoprosencephaly. Nat Genet 1996, 14:357-360.
7. Ming JE, Kaupas ME, Roessler E, Brunner HG, Golabi M, Tekin M, Stratton RF, Sujansky E, Bale SJ, Muenke M: Mutations in PATCHED-1, the receptor for SONIC HEDGEHOG, are associated with holoprosencephaly. Hum Genet 2002, 110:297-301.
8. Muenke M, Beachy PA: Holoprosencephaly. In Metabolic and Molecular Bases of inherited Disease. Edited by Scriver CR, Beaudet AL, Sly WS, Valle D, Childs B, Hilds B, Kinzler KW, Vogelstein B. New York: McGraw-Hill; 2001:6203-6230.
9. Oro AE, Higgins KM, Hu Z, Bonifas JM, Epstein EH, Scott MP: Basal cell carcinomas in mice overexpressing sonic hedgehog. Science 1997, 276:817-821.
10. Dahmane N, Lee J, Robin P, Heller P, Ruiz i Altaba: Activation of the transcription factor Gli1 and the Sonic hedgehog signalling pathway in skin tumours. Nature 1997, 389:876-881.
11. Nilsson M, Unden AB, Krause D, Malmqwist U, Raza K, Zaphiropoulos PG, Toftgard R: Induction of basal cell carcinomas and trichoepitheliomas in mice overexpressing GLI-1. Proc Natl Acad Sci USA 2000, 97:3438-3443.
12. Grachtchouk M, Mo R, Yu S, Zhang X, Sasaki H, Hui C-c, Dlugosz AA: Basal cell carcinomas in mice overexpressing Gli2 in skin. Nat Genet 2000, 24:216-217.
13. Ruiz i Altaba A., Sanchez P, Dahmane N: Gli and hedgehog in cancer: tumours, embryos and stem cells. Nat Rev Cancer 2002, 2:361-372.
14. Taylor MD, Liu L, Raffel C, Hui C-c, Mainproze TG, Zhang X, Agatep R, Chiappa S, Gao L, Lowrance A, et al.: Mutations in SUFU predispose to medulloblastoma. Nat Genet 2002, 31:306-310.
15. Goodrich LV, Milenkovic L, Higgins KM, Scott MP. Altered neural cell fates and medulloblastoma in mouse patched mutants. Science 1997, 277:1109-1113.
16. Raffel C, Jenkins RB, Frederick I, Hebrink D, Alderete B, Fults DW, James CD: Sporadic medulloblastomas contains PTCH mutations. Cancer Res 1997, 57:842-845.
17. Wolter M, Reifenberger J, Sommer C, Ruzicka T, Reifenberger G: Mutations in the human homologue of the Drosophila segment polarity gene patched (PTCH) in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system. Cancer Res 1997, 57:2581-2585.
18. Dahmane N, Sanchez P, Gitton Y, Palma V, Sun T, Beyna M, Weiner H, Ruiz i Altaba A: The Sonic Hedgehog-Gli pathway regulates dorsal brain growth and tumorigenesis. Development 2001, 128:5201-5212.
19. Pomeroy SL, Tamayo P, Gaasenbeek M, Sturla LM, Angelo M, McLaughlin ME, Kim JY, Goumnerova LC, Black PM, Lau C, et al.: Prediction of central nervous system embryonal tumour outcome based on gene expression. Nature 2002, 415:436-442.
20. Tsuboi K, Shults CW: Intrastriatal injection of sonic hedgehog reduces behavioral impairment in a rat model of Parkinson's disease. Exp Neurol 2002, 173:95-104.
21. Wang MZ, Jin P, Bumcrot DA, Marigo V, McMahon AP, Wang EA, Woolf T, Pang K: Induction of dopaminergic neuron phenotype in the midbrain by Sonic hedgehog protein. Nat Med 1995, 1:1184-1188.
22. Hynes M, Porter JA, Chiang C, Chang D, Tessier-Lavigne M, Beachy PA: Induction of midbrain dopaminergic neurons by Sonic hedgehog. Neuron 1995, 15:35-44.
23. Miao N, Wang M, Ott JA, D'Alessandro JS, Woolf TM, Bumcrot DA, Mahanthappa NK, Pang K: Sonic hedgehog promotes the survival of specific CNS neuron populations and protects these cells from toxic insult in vitro. J Neurosci 1997, 17:5891-5899.
24. Pepinsky RB, Shapiro RI, Wang S, Chakraborty A, Gill A, Lepage D, Wen D, Rayhorn, Horan GSB, Taylor FR, et al.: Long-acting form of sonic hedgehog with improved pharmacokinetic and pharmacodynamic properties are efficacious in a nerve injury model. J Pharm Sci 2002, 91:371-387.
25. Chen JK, Taipale J, Young KE, Maiti T, Beachy PA: Small molecule modulation of Smoothened activity. Proc Natl Acad Sci USA 2002, 99:14071-14076.
26. Ericson J, Morton S, Kawakami A, Roelink H, Jessel TM: Two critical periods of sonic hedgehog signaling required for the specification of motor neuron identity. Cell 1996, 87:661-673.
27. Incardona JP, Gaffield W, Kapur RP, Roelink H: The teratogenic Veratrum alkaloid cyclopamine inhibits sonic hedgehog signal transduction. Development 1998, 125:3553-3562.
28. Cooper MK, Porter JA, Young KE, Beachy PA: Teratogen-mediated inhibition of target tissue response to Shh signaling. Science 1998, 280:1603-1607.
29. Taipale J, Chen JK, Cooper MK, Wang B, Mann RK, Milenkovic L, Scott MP, Beachy PA: Effects of oncogenic mutations in Smoothened and Patched can be reversed by cyclopamine. Nature 2000, 406:1005-1009.
30. Fan CM, Porter JA, Chiang C, Chang DT, Beachy PA, Tessier-Lavigne M: Long-range sclerotome induction by sonic hedgehog: direct role of the amino-terminal cleavage product and modulation by the cyclic AMP signaling pathway. Cell 1995, 81:457-465.
31. Ruiz i Altaba: Gli proteins encode context-dependent positive and negative functions: implication for development and diseases. Development 1999, 126:3205-3216.
32. Berman DM, Karhadkar SS, Hallahan AR, Pritchard JI, Eberhart CG, Watkins DN, Chen JK, Cooper MK, Taiplae J, Olson JM, Beachy PA: Medulloblastoma growth inhibition by hedgehog pathway blockade. Science 2002, 297:1559-1561.
33. Williams JA, Guicherit OM, Zaharian BI, Xu Y, Chai L, Gatchalian C, Porter JA, Rubin LL, Wang FY: Identification of novel inhibitors of the hedgehog signaling pathway: effects on basal cell carcinoma-like lesions. Proc Natl Acad Sci USA 2002, in press.
34. Sasaki H, Nishizaki Y, Hui C, Nakafuku M, Kondoh H: Regulation of Gli2 and Gli3 activities by an amino-terminal repressor domain: implication of Gli2 and Gli3 as primary mediators of Shh signaling. Development 1999, 126:3915-3924.
35. Shin SH, Kogerman P, Lindstrom E, Toftgard R, Biesecker LG: Gli3 mutations in human disorders mimic Drosophila cubitus interruptus protein functions and localizations. Proc Natl Acad Sci USA 1999, 96:2880-2884.
36. Derossi D, Chassing G, Prochiantz A: Trojan peptides: the penetratin system for intracellular delivery. Trends Cell Biol 1998, 8:84-87.