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Lee Rubin, Ph.D.

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Research
The research activities and funding listed below are automatically derived from NIH ExPORTER and other sources, which might result in incorrect or missing items. Faculty can login to make corrections and additions.
  1. RC4DK090781 (MELTON, DOUGLAS A) Sep 25, 2010 - Sep 30, 2013
    NIH/NIDDK
    Identification of Genes and Compounds That Control Beta Cell Replication
    Role: Co-Principal Investigator
  2. P01NS066888 (VAN VACTOR, DAVID L.) Aug 1, 2010 - Jul 31, 2015
    NIH/NINDS
    Genetic Modeling for SMA Therapeutic Pathways
    Role: Co-Principal Investigator

Bibliographic
Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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  1. Rodriguez-Muela N, Parkhitko A, Grass T, Gibbs RM, Norabuena EM, Perrimon N, Singh R, Rubin LL. Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes. J Clin Invest. 2018 Jun 11. PMID: 29672276.
    View in: PubMed
  2. Christiansen EM, Yang SJ, Ando DM, Javaherian A, Skibinski G, Lipnick S, Mount E, O'Neil A, Shah K, Lee AK, Goyal P, Fedus W, Poplin R, Esteva A, Berndl M, Rubin LL, Nelson P, Finkbeiner S. In Silico Labeling: Predicting Fluorescent Labels in Unlabeled Images. Cell. 2018 Apr 19; 173(3):792-803.e19. PMID: 29656897.
    View in: PubMed
  3. Ordureau A, Paulo JA, Zhang W, Ahfeldt T, Zhang J, Cohn EF, Hou Z, Heo JM, Rubin LL, Sidhu SS, Gygi SP, Harper JW. Dynamics of PARKIN-Dependent Mitochondrial Ubiquitylation in Induced Neurons and Model Systems Revealed by Digital Snapshot Proteomics. Mol Cell. 2018 Apr 19; 70(2):211-227.e8. PMID: 29656925.
    View in: PubMed
  4. Paik EJ, O'Neil AL, Ng SY, Sun C, Rubin LL. Using intracellular markers to identify a novel set of surface markers for live cell purification from a heterogeneous hIPSC culture. Sci Rep. 2018 Jan 16; 8(1):804. PMID: 29339826.
    View in: PubMed
  5. Feinberg K, Kolaj A, Wu C, Grinshtein N, Krieger JR, Moran MF, Rubin LL, Miller FD, Kaplan DR. A neuroprotective agent that inactivates prodegenerative TrkA and preserves mitochondria. J Cell Biol. 2017 11 06; 216(11):3655-3675. PMID: 28877995.
    View in: PubMed
  6. Tripathi P, Rodriguez-Muela N, Klim JR, de Boer AS, Agrawal S, Sandoe J, Lopes CS, Ogliari KS, Williams LA, Shear M, Rubin LL, Eggan K, Zhou Q. Reactive Astrocytes Promote ALS-like Degeneration and Intracellular Protein Aggregation in Human Motor Neurons by Disrupting Autophagy through TGF-ß1. Stem Cell Reports. 2017 08 08; 9(2):667-680. PMID: 28712846.
    View in: PubMed
  7. Palomo V, Perez DI, Roca C, Anderson C, Rodríguez-Muela N, Perez C, Morales-Garcia JA, Reyes JA, Campillo NE, Perez-Castillo AM, Rubin LL, Timchenko L, Gil C, Martinez A. Subtly Modulating Glycogen Synthase Kinase 3 ß: Allosteric Inhibitor Development and Their Potential for the Treatment of Chronic Diseases. J Med Chem. 2017 Jun 22; 60(12):4983-5001. PMID: 28548834.
    View in: PubMed
  8. Rodriguez-Muela N, Litterman NK, Norabuena EM, Mull JL, Galazo MJ, Sun C, Ng SY, Makhortova NR, White A, Lynes MM, Chung WK, Davidow LS, Macklis JD, Rubin LL. Single-Cell Analysis of SMN Reveals Its Broader Role in Neuromuscular Disease. Cell Rep. 2017 02 07; 18(6):1484-1498. PMID: 28178525.
    View in: PubMed
  9. d'Ydewalle C, Ramos DM, Pyles NJ, Ng SY, Gorz M, Pilato CM, Ling K, Kong L, Ward AJ, Rubin LL, Rigo F, Bennett CF, Sumner CJ. The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy. Neuron. 2017 Jan 04; 93(1):66-79. PMID: 28017471.
    View in: PubMed
  10. Rigamonti A, Repetti GG, Sun C, Price FD, Reny DC, Rapino F, Weisinger K, Benkler C, Peterson QP, Davidow LS, Hansson EM, Rubin LL. Large-Scale Production of Mature Neurons from Human Pluripotent Stem Cells in a Three-Dimensional Suspension Culture System. Stem Cell Reports. 2016 06 14; 6(6):993-1008. PMID: 27304920; PMCID: PMC4912437.
  11. Ahfeldt T, Litterman NK, Rubin LL. Studying human disease using human neurons. Brain Res. 2017 02 01; 1656:40-48. PMID: 27060768.
    View in: PubMed
  12. Walker RG, Poggioli T, Katsimpardi L, Buchanan SM, Oh J, Wattrus S, Heidecker B, Fong YW, Rubin LL, Ganz P, Thompson TB, Wagers AJ, Lee RT. Biochemistry and Biology of GDF11 and Myostatin: Similarities, Differences, and Questions for Future Investigation. Circ Res. 2016 Apr 01; 118(7):1125-41; discussion 1142. PMID: 27034275; PMCID: PMC4818972 [Available on 04/01/17].
  13. Brennand KJ, Marchetto MC, Benvenisty N, Brüstle O, Ebert A, Izpisua Belmonte JC, Kaykas A, Lancaster MA, Livesey FJ, McConnell MJ, McKay RD, Morrow EM, Muotri AR, Panchision DM, Rubin LL, Sawa A, Soldner F, Song H, Studer L, Temple S, Vaccarino FM, Wu J, Vanderhaeghen P, Gage FH, Jaenisch R. Creating Patient-Specific Neural Cells for the In Vitro Study of Brain Disorders. Stem Cell Reports. 2015 Dec 08; 5(6):933-945. PMID: 26610635.
    View in: PubMed
  14. Ng SY, Soh BS, Rodriguez-Muela N, Hendrickson DG, Price F, Rinn JL, Rubin LL. Genome-wide RNA-Seq of Human Motor Neurons Implicates Selective ER Stress Activation in Spinal Muscular Atrophy. Cell Stem Cell. 2015 Nov 05; 17(5):569-84. PMID: 26321202; PMCID: PMC4839185 [Available on 11/05/16].
  15. Xue R, Lynes MD, Dreyfuss JM, Shamsi F, Schulz TJ, Zhang H, Huang TL, Townsend KL, Li Y, Takahashi H, Weiner LS, White AP, Lynes MS, Rubin LL, Goodyear LJ, Cypess AM, Tseng YH. Clonal analyses and gene profiling identify genetic biomarkers of the thermogenic potential of human brown and white preadipocytes. Nat Med. 2015 Jul; 21(7):760-8. PMID: 26076036; PMCID: PMC4496292.
  16. Katsimpardi L, Rubin LL. Young systemic factors as a medicine for age-related neurodegenerative diseases. Neurogenesis (Austin). 2015; 2(1):e1004971. PMID: 27502604; PMCID: PMC4973601.
  17. Schlaeger TM, Daheron L, Brickler TR, Entwisle S, Chan K, Cianci A, DeVine A, Ettenger A, Fitzgerald K, Godfrey M, Gupta D, McPherson J, Malwadkar P, Gupta M, Bell B, Doi A, Jung N, Li X, Lynes MS, Brookes E, Cherry AB, Demirbas D, Tsankov AM, Zon LI, Rubin LL, Feinberg AP, Meissner A, Cowan CA, Daley GQ. A comparison of non-integrating reprogramming methods. Nat Biotechnol. 2015 Jan; 33(1):58-63. PMID: 25437882; PMCID: PMC4329913.
  18. Wainger BJ, Buttermore ED, Oliveira JT, Mellin C, Lee S, Saber WA, Wang AJ, Ichida JK, Chiu IM, Barrett L, Huebner EA, Bilgin C, Tsujimoto N, Brenneis C, Kapur K, Rubin LL, Eggan K, Woolf CJ. Modeling pain in vitro using nociceptor neurons reprogrammed from fibroblasts. Nat Neurosci. 2015 Jan; 18(1):17-24. PMID: 25420066; PMCID: PMC4429606.
  19. Woodard CM, Campos BA, Kuo SH, Nirenberg MJ, Nestor MW, Zimmer M, Mosharov EV, Sulzer D, Zhou H, Paull D, Clark L, Schadt EE, Sardi SP, Rubin L, Eggan K, Brock M, Lipnick S, Rao M, Chang S, Li A, Noggle SA. iPSC-derived dopamine neurons reveal differences between monozygotic twins discordant for Parkinson's disease. Cell Rep. 2014 Nov 20; 9(4):1173-82. PMID: 25456120; PMCID: PMC4255586.
  20. Blum B, Roose AN, Barrandon O, Maehr R, Arvanites AC, Davidow LS, Davis JC, Peterson QP, Rubin LL, Melton DA. Reversal of ß cell de-differentiation by a small molecule inhibitor of the TGFß pathway. Elife. 2014 Sep 16; 3:e02809. PMID: 25233132; PMCID: PMC4204634.
  21. Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 08; 345(6197):688-93. PMID: 25104390.
    View in: PubMed
  22. Cherry JJ, Kobayashi DT, Lynes MM, Naryshkin NN, Tiziano FD, Zaworski PG, Rubin LL, Jarecki J. Assays for the identification and prioritization of drug candidates for spinal muscular atrophy. Assay Drug Dev Technol. 2014 Aug; 12(6):315-41. PMID: 25147906; PMCID: PMC4142828.
  23. Ichida JK, Tcw J, Williams LA, Carter AC, Shi Y, Moura MT, Ziller M, Singh S, Amabile G, Bock C, Umezawa A, Rubin LL, Bradner JE, Akutsu H, Meissner A, Eggan K. Notch inhibition allows oncogene-independent generation of iPS cells. Nat Chem Biol. 2014 Aug; 10(8):632-639. PMID: 24952596; PMCID: PMC4310751.
  24. Gould SE, Low JA, Marsters JC, Robarge K, Rubin LL, de Sauvage FJ, Sutherlin DP, Wong H, Yauch RL. Discovery and preclinical development of vismodegib. Expert Opin Drug Discov. 2014 Aug; 9(8):969-84. PMID: 24857041.
    View in: PubMed
  25. Katsimpardi L, Litterman NK, Schein PA, Miller CM, Loffredo FS, Wojtkiewicz GR, Chen JW, Lee RT, Wagers AJ, Rubin LL. Vascular and neurogenic rejuvenation of the aging mouse brain by young systemic factors. Science. 2014 May 09; 344(6184):630-4. PMID: 24797482; PMCID: PMC4123747.
  26. Sen A, Dimlich DN, Guruharsha KG, Kankel MW, Hori K, Yokokura T, Brachat S, Richardson D, Loureiro J, Sivasankaran R, Curtis D, Davidow LS, Rubin LL, Hart AC, Van Vactor D, Artavanis-Tsakonas S. Genetic circuitry of Survival motor neuron, the gene underlying spinal muscular atrophy. Proc Natl Acad Sci U S A. 2013 Jun 25; 110(26):E2371-80. PMID: 23757500; PMCID: PMC3696827.
  27. Yang YM, Gupta SK, Kim KJ, Powers BE, Cerqueira A, Wainger BJ, Ngo HD, Rosowski KA, Schein PA, Ackeifi CA, Arvanites AC, Davidow LS, Woolf CJ, Rubin LL. A small molecule screen in stem-cell-derived motor neurons identifies a kinase inhibitor as a candidate therapeutic for ALS. Cell Stem Cell. 2013 Jun 06; 12(6):713-26. PMID: 23602540; PMCID: PMC3707511.
  28. Ding Q, Lee YK, Schaefer EA, Peters DT, Veres A, Kim K, Kuperwasser N, Motola DL, Meissner TB, Hendriks WT, Trevisan M, Gupta RM, Moisan A, Banks E, Friesen M, Schinzel RT, Xia F, Tang A, Xia Y, Figueroa E, Wann A, Ahfeldt T, Daheron L, Zhang F, Rubin LL, Peng LF, Chung RT, Musunuru K, Cowan CA. A TALEN genome-editing system for generating human stem cell-based disease models. Cell Stem Cell. 2013 Feb 07; 12(2):238-51. PMID: 23246482; PMCID: PMC3570604.
  29. Wang Y, Davidow L, Arvanites AC, Blanchard J, Lam K, Xu K, Oza V, Yoo JW, Ng JM, Curran T, Rubin LL, McMahon AP. Glucocorticoid compounds modify smoothened localization and hedgehog pathway activity. Chem Biol. 2012 Aug 24; 19(8):972-82. PMID: 22921064; PMCID: PMC3724998.
  30. Hayhurst M, Wagner AK, Cerletti M, Wagers AJ, Rubin LL. A cell-autonomous defect in skeletal muscle satellite cells expressing low levels of survival of motor neuron protein. Dev Biol. 2012 Aug 15; 368(2):323-34. PMID: 22705478; PMCID: PMC3851302.
  31. Wang Y, Arvanites AC, Davidow L, Blanchard J, Lam K, Yoo JW, Coy S, Rubin LL, McMahon AP. Selective identification of hedgehog pathway antagonists by direct analysis of smoothened ciliary translocation. ACS Chem Biol. 2012 Jun 15; 7(6):1040-8. PMID: 22554036; PMCID: PMC3905677.
  32. Annes JP, Ryu JH, Lam K, Carolan PJ, Utz K, Hollister-Lock J, Arvanites AC, Rubin LL, Weir G, Melton DA. Adenosine kinase inhibition selectively promotes rodent and porcine islet ß-cell replication. Proc Natl Acad Sci U S A. 2012 Mar 06; 109(10):3915-20. PMID: 22345561; PMCID: PMC3309788.
  33. Chen PC, Gaisina IN, El-Khodor BF, Ramboz S, Makhortova NR, Rubin LL, Kozikowski AP. Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, that Prolongs the Median Survival Time of ?7 SMA KO Mouse Model of Spinal Muscular Atrophy. ACS Chem Neurosci. 2012 Jan 18; 3(1):5-11. PMID: 22348181.
    View in: PubMed
  34. Makhortova NR, Hayhurst M, Cerqueira A, Sinor-Anderson AD, Zhao WN, Heiser PW, Arvanites AC, Davidow LS, Waldon ZO, Steen JA, Lam K, Ngo HD, Rubin LL. A screen for regulators of survival of motor neuron protein levels. Nat Chem Biol. 2011 Jun 19; 7(8):544-52. PMID: 21685895; PMCID: PMC3236614.
  35. Rubin LL, Haston KM. Stem cell biology and drug discovery. BMC Biol. 2011 Jun 07; 9:42. PMID: 21649940; PMCID: PMC3110139.
  36. Tang T, Tang JY, Li D, Reich M, Callahan CA, Fu L, Yauch RL, Wang F, Kotkow K, Chang KS, Shpall E, Wu A, Rubin LL, Marsters JC, Epstein EH, Caro I, de Sauvage FJ. Targeting superficial or nodular Basal cell carcinoma with topically formulated small molecule inhibitor of smoothened. Clin Cancer Res. 2011 May 15; 17(10):3378-87. PMID: 21558397; PMCID: PMC3113453.
  37. Haramati S, Chapnik E, Sztainberg Y, Eilam R, Zwang R, Gershoni N, McGlinn E, Heiser PW, Wills AM, Wirguin I, Rubin LL, Misawa H, Tabin CJ, Brown R, Chen A, Hornstein E. miRNA malfunction causes spinal motor neuron disease. Proc Natl Acad Sci U S A. 2010 Jul 20; 107(29):13111-6. PMID: 20616011; PMCID: PMC2919953.
  38. Rihel J, Prober DA, Arvanites A, Lam K, Zimmerman S, Jang S, Haggarty SJ, Kokel D, Rubin LL, Peterson RT, Schier AF. Zebrafish behavioral profiling links drugs to biological targets and rest/wake regulation. Science. 2010 Jan 15; 327(5963):348-51. PMID: 20075256; PMCID: PMC2830481.
  39. Ichida JK, Blanchard J, Lam K, Son EY, Chung JE, Egli D, Loh KM, Carter AC, Di Giorgio FP, Koszka K, Huangfu D, Akutsu H, Liu DR, Rubin LL, Eggan K. A small-molecule inhibitor of tgf-Beta signaling replaces sox2 in reprogramming by inducing nanog. Cell Stem Cell. 2009 Nov 06; 5(5):491-503. PMID: 19818703; PMCID: PMC3335195.
  40. Robarge KD, Brunton SA, Castanedo GM, Cui Y, Dina MS, Goldsmith R, Gould SE, Guichert O, Gunzner JL, Halladay J, Jia W, Khojasteh C, Koehler MF, Kotkow K, La H, Lalonde RL, Lau K, Lee L, Marshall D, Marsters JC, Murray LJ, Qian C, Rubin LL, Salphati L, Stanley MS, Stibbard JH, Sutherlin DP, Ubhayaker S, Wang S, Wong S, Xie M. GDC-0449-a potent inhibitor of the hedgehog pathway. Bioorg Med Chem Lett. 2009 Oct 01; 19(19):5576-81. PMID: 19716296.
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  41. Brunton SA, Stibbard JH, Rubin LL, Guicherit OM, Kruse LI, Price S, di Lucrezia R, MacKinnon CH, Avery A, Park Y, Buxton D, Boyd EA. Potent agonists of the Hedgehog signaling pathway. Bioorg Med Chem Lett. 2009 Aug 01; 19(15):4308-11. PMID: 19500978.
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  42. Chen S, Borowiak M, Fox JL, Maehr R, Osafune K, Davidow L, Lam K, Peng LF, Schreiber SL, Rubin LL, Melton D. A small molecule that directs differentiation of human ESCs into the pancreatic lineage. Nat Chem Biol. 2009 Apr; 5(4):258-65. PMID: 19287398.
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  43. Yauch RL, Gould SE, Scales SJ, Tang T, Tian H, Ahn CP, Marshall D, Fu L, Januario T, Kallop D, Nannini-Pepe M, Kotkow K, Marsters JC, Rubin LL, de Sauvage FJ. A paracrine requirement for hedgehog signalling in cancer. Nature. 2008 Sep 18; 455(7211):406-10. PMID: 18754008.
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  44. Rubin LL. Stem cells and drug discovery: the beginning of a new era? Cell. 2008 Feb 22; 132(4):549-52. PMID: 18295572.
    View in: PubMed
  45. Brunton SA, Stibbard JH, Rubin LL, Kruse LI, Guicherit OM, Boyd EA, Price S. Potent inhibitors of the hedgehog signaling pathway. J Med Chem. 2008 Mar 13; 51(5):1108-10. PMID: 18275133.
    View in: PubMed
  46. Rubin LL, de Sauvage FJ. Targeting the Hedgehog pathway in cancer. Nat Rev Drug Discov. 2006 Dec; 5(12):1026-33. PMID: 17139287.
    View in: PubMed
  47. Deshpande DM, Kim YS, Martinez T, Carmen J, Dike S, Shats I, Rubin LL, Drummond J, Krishnan C, Hoke A, Maragakis N, Shefner J, Rothstein JD, Kerr DA. Recovery from paralysis in adult rats using embryonic stem cells. Ann Neurol. 2006 Jul; 60(1):32-44. PMID: 16802299.
    View in: PubMed
  48. Soundararajan P, Miles GB, Rubin LL, Brownstone RM, Rafuse VF. Motoneurons derived from embryonic stem cells express transcription factors and develop phenotypes characteristic of medial motor column neurons. J Neurosci. 2006 Mar 22; 26(12):3256-68. PMID: 16554476.
    View in: PubMed
  49. Dellovade T, Romer JT, Curran T, Rubin LL. The hedgehog pathway and neurological disorders. Annu Rev Neurosci. 2006; 29:539-63. PMID: 16776596.
    View in: PubMed
  50. Paladini RD, Saleh J, Qian C, Xu GX, Rubin LL. Modulation of hair growth with small molecule agonists of the hedgehog signaling pathway. J Invest Dermatol. 2005 Oct; 125(4):638-46. PMID: 16185261.
    View in: PubMed
  51. Schnapp E, Kragl M, Rubin L, Tanaka EM. Hedgehog signaling controls dorsoventral patterning, blastema cell proliferation and cartilage induction during axolotl tail regeneration. Development. 2005 Jul; 132(14):3243-53. PMID: 15983402.
    View in: PubMed
  52. Dvash T, Mayshar Y, Darr H, McElhaney M, Barker D, Yanuka O, Kotkow KJ, Rubin LL, Benvenisty N, Eiges R. Temporal gene expression during differentiation of human embryonic stem cells and embryoid bodies. Hum Reprod. 2004 Dec; 19(12):2875-83. PMID: 15375076.
    View in: PubMed
  53. Romer JT, Kimura H, Magdaleno S, Sasai K, Fuller C, Baines H, Connelly M, Stewart CF, Gould S, Rubin LL, Curran T. Suppression of the Shh pathway using a small molecule inhibitor eliminates medulloblastoma in Ptc1(+/-)p53(-/-) mice. Cancer Cell. 2004 Sep; 6(3):229-40. PMID: 15380514.
    View in: PubMed
  54. Lin LF, Rubin LL, Xu M. A non-peptidyl neurotrophic small molecule for midbrain dopaminergic neurons. J Neurochem. 2004 Jun; 89(6):1387-95. PMID: 15189341.
    View in: PubMed
  55. Kessaris N, Jamen F, Rubin LL, Richardson WD. Cooperation between sonic hedgehog and fibroblast growth factor/MAPK signalling pathways in neocortical precursors. Development. 2004 Mar; 131(6):1289-98. PMID: 14960493.
    View in: PubMed
  56. Gabay L, Lowell S, Rubin LL, Anderson DJ. Deregulation of dorsoventral patterning by FGF confers trilineage differentiation capacity on CNS stem cells in vitro. Neuron. 2003 Oct 30; 40(3):485-99. PMID: 14642274.
    View in: PubMed
  57. Machold R, Hayashi S, Rutlin M, Muzumdar MD, Nery S, Corbin JG, Gritli-Linde A, Dellovade T, Porter JA, Rubin LL, Dudek H, McMahon AP, Fishell G. Sonic hedgehog is required for progenitor cell maintenance in telencephalic stem cell niches. Neuron. 2003 Sep 11; 39(6):937-50. PMID: 12971894.
    View in: PubMed
  58. Williams JA, Guicherit OM, Zaharian BI, Xu Y, Chai L, Wichterle H, Kon C, Gatchalian C, Porter JA, Rubin LL, Wang FY. Identification of a small molecule inhibitor of the hedgehog signaling pathway: effects on basal cell carcinoma-like lesions. Proc Natl Acad Sci U S A. 2003 Apr 15; 100(8):4616-21. PMID: 12679522; PMCID: PMC153604.
  59. 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 Nov 06; 1(2):10. PMID: 12437772; PMCID: PMC137065.
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