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Expert Opinion on Emerging Drugs Volume 10, 2005 - Issue 2
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Review

Emerging chemotherapeutic strategies for Huntington’s disease

Hoon RyuBoston University School of Medicine Edith Nourse Rogers Veterans Administration Medical Center, Experimental Neuropathology Unit and Translational Therapeutics Laboratory, GRECC Unit, Building 18, Bedford, Massachusetts 01730, USATel: +1 781 687 2908; Fax: +1 781 687 3515;E-mail: [email protected]
&
Robert J FerranteBoston University School of Medicine Edith Nourse Rogers Veterans Administration Medical Center, Experimental Neuropathology Unit and Translational Therapeutics Laboratory, GRECC Unit, Building 18, Bedford, Massachusetts 01730, USATel: +1 781 687 2908; Fax: +1 781 687 3515;E-mail: [email protected]
Pages 345-363 | Published online: 10 May 2005

Bibliography

  • KREMER BP, GOLDBERG S, ANDREW J et al.: A worldwide study of the Huntington's disease mutation — the sensitivity and specificity of measuring GAG repeats. N Engl. J. Med. (1994) 330:1401–1406.
  • MYERS R, MACDONALD M, KOROSHETZ W et al.: De novo expansion of a (CAG)n repeat in sporadic Huntington's disease. Nat. Genet. (1993) 5:168–173.
  • PAULSEN JS, ZHAO H, STOUT JC et al.: Clinical markers of early disease in persons near onset of Huntington's disease. Neurology (2001) 57:658–662.
  • HELDER DI, KAPTEIN AA, VAN KEMPEN GM, VAN HOUWELINGEN JC, ROOS RA: Impact of Huntington's disease on quality of life. Mov. Disord. (2001) 16:325–330.
  • FERRANTE RJ, KOWALL NW, BEAL MF et al.: Selective sparing of a class of striatal neurons in Huntington's disease. Science (1985) 230:561–563.
  • HERSCH SM, ROSAS HD, FERRANTE RJ: Neuropathology and pathophysiology of Huntington's disease, in movement disorders. In: Neurologic Principles and Practice. Koller W (Ed.), McGraw-Hill, New York, USA (2004):503–526.
  • FERRANTE RJ, KOWALL NW, RI CHARDSON EP Jr : Proliferative and degenerative changes in striatal spiny neurons in Huntington's disease: a combined study using the section-Golgi method and calbindin D28k immunocytochemistry. J. Neurosci. (1991) 11:3877–3887.
  • GRAVELAND GA, WILLIAMS RS, DIFIGLIA MA: Evidence for degenerative and regenerative changes in neostriatal spiny neurons in Huntington's disease. Science (1985) 227:770–773.
  • HUNTINGTON'S DISEASE COLLABORATIVE RESEARCH GROUP: A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell (1993)72:971–983.
  • SZEBENYI G, MORFINI GA, BABCOCK A et al.: Neuropathogenic forms of huntingtin and androgen receptor inhibit fast axonal transport. Neuron (2003) 40:41–52.
  • TRUSHINA E, HELDEBRANT MP, PEREZ-TERZIC CM et al.: Microtubule destabilization and nuclear entry are sequential steps leading to toxicity in Huntington's disease. Proc. NatL Acad. Sci. USA (2003) 100:12171–12176.
  • GAUTHIER LR, CHARRIN BC, BORRELL-PAGES M et al.: Huntingtin controls neurotrophic support and survival of neurons by enhancing BDNF vesicular transport along microtubules. Cell (2004) 118:127–138.
  • ROSS CA: Huntington's disease: new paths to pathogenesis. Cell (2004) 118(1):4–7.
  • ROBITAILLE Y, LOPES-CENDES I, BECHER M, ROULEAU G, CLARK AW: The neuropathology of CAG repeat diseases: review and update of genetic and molecular features. Brain PathoL (1997) 7:901–926.
  • ROSS CA: Intranuclear neuronal inclusions: a common pathogenic mechanism for glutamine-repeat neurodegenerative diseases? Neuron (1997) 19:1147–1150.
  • ROSENBLATT A, RANEN N, NANCE M, PAULSEN J: A physician's guide to the management of Huntington's Disease (2nd edition), Huntington's Disease Society of America, New York, USA (1999).
  • GROUP HS: Unified Huntington's disease rating scale: reliability and consistency. Huntington Study Group. Mov. Disord. (1996) 11:136–142.
  • JENKINS BG, KOROSHETZ WJ, BEAL MF, ROSEN BR: Evidence for impairment of energy metabolism in vivo in Huntington's disease using localized 1H NMR spectroscopy. Neurology (1993) 43:2689–2695.
  • KIEBURTZ K, FEIGIN A, MCDERMOTT M et al.: A controlled trial of remacemide hydrochloride in Huntington's disease. Mov. Disord. (1996) 11:273–277.
  • GROUP HS: Safety and tolerability of thefree-radical scavenger OPC-14117 in Huntington's disease. Neurology (1998) 50:1366–1373.
  • GROUP HS: A randomized, placebo-controlled trial of coenzyme Q10 and remacemide in Huntington's disease. Neurology (2001) 57:397–404.
  • PEYSER CE, FOLSTEIN M, CHASE GA et al.: Trial of D-alpha-tocopherol in Huntington's disease. Am. J. Psychiatry (1995) 152:1771–1775.
  • RANEN NG, PEYSER CE, COYLE JT et al.: A controlled trial of idebenone in Huntington's disease. Mov. Disord. (1996) 11:549–554.
  • KREMER B, CLARK C, HARDY M, ALMQVIST E, RAYMOND L, HAYDEN M: Lamotrigine does not retard the progression of Huntington's disease. WFN Working Group on Huntington's Disease, (1997):34.
  • ROSAS HD, KOROSHETZ WJ, JENKINS BG et al.: Riluzole therapy in Huntington's disease (HD). Mov. Disord. (1999) 14:326–330.
  • SMITH RA, KELSO GF, JAMES AM, MURPHY MP: Targeting coenzyme Q derivatives to mitochondria. Methods Enqmol. (2004) 382:45–67.
  • SHULTS CW, BEAL MF, SONG D, FONTAINE D: Pilot trial of high dosages of coenzyme Q10 in patients with Parkinson's disease. Exp. NeuroL (2004) 188:491–494.
  • YAMAMOTO A. LUCAS JJ, HEN R: Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease. Cell (2000) 101:57–66.
  • BEAL MF, FERRANTE RJ: Experimentaltherapeutics in transgenic mouse models of Huntington's disease. Nat. Rev. Neurosci. (2004) 5:373–384.
  • HERSCH SM, FERRANTE RJ: Translating therapies for Huntington's disease from genetic animal models to clinical trials. Neurokc (2004) 1:298–306.
  • PERSICHETTI F, AMBROSE CM, GE P et al.: Normal and expanded Huntington's disease gene alleles produce distinguishable proteins due to translation across the CAG repeat. Mol. Med. (1995) 1:374–383.
  • FERRANTE RJ, GUTEKUNST CA, PERSICHETTI F et al.: Heterogeneous topographic and cellular distribution of huntingtin expression in the normal human neostriatum. J. Neurosci. (1997) 17: 3052–3063.
  • DIFIGLIA M, SAPP E, CHASE KO et al.:Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons. Neuron (1995) 14:1075–1081.
  • ZUCCATO C, CIAMMOLA A, RIGAMONTI D et al.: Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease. Science (2001) 293:493–498.
  • ZHANG Y, LI M, DROZDA M et al.: Depletion of wild-type huntingtin in mouse models of neurologic diseases. J. Neurochem. (2003) 87:102–106.
  • WELLINGTON CL, BRINKMAN RR, O'KUSKY JR, HAYDEN MR: Toward understanding the molecular pathology of Huntington's disease. Brain PathoL (1997) 7:979–1002.
  • PAULSON HL: Protein fate in neurodegenerative proteinopathies: polyglutamine diseases join the (mis)fold. Am. J. Hum. Genet. (1999) 64:339–345.
  • JANA NR, ZEMSKOV EA, WANG G, NUKINA N: Altered proteasomal function due to the expression of polyglutamine-expanded truncated N-terminal huntingtin induces apoptosis by caspase activation through mitochondrial cytochrome c release. Hum. MoL Genet. (2001) 10:1049–1059.
  • JANA NR, TANAKA M, WANG G, NUKINA N: Polyglutamine length-dependent interaction of Hsp40 and Hsp70 family chaperones with truncated N-terminal huntingtin: their role in suppression of aggregation and cellular toxicity. Hum. MoL Genet. (2000) 9:2009–2018.
  • CHAT Y, KOPPENHAFER SL, SHOESMITH SJ, PEREZ MK, PAULSON HL: Evidence for proteasome involvement in polyglutamine disease: localization to nuclear inclusions in SCA3/ MJD and suppression of polyglutamine aggregation in vitro. Hum. MoL Genet. (1999) 8:673–682.
  • CHA JH: Transcriptional dysregulation in Huntington's disease. Trends Neurosci. (2000) 23(9):387–392.
  • LUTHI-CARTER R, APOSTOL BL, DUNAH AW et al.: Complex alteration of NMDA receptors in transgenic Huntington's disease mouse brain: analysis of mRNA and protein expression, plasma membrane association, interacting proteins, and phosphorylation. NeurobioL Dis. (2003) 14:624–636.
  • PREISINGER E, JORDAN BM, KAZANTSEV A, HOUSMAN D: Evidence for a recruitment and sequestration mechanism in Huntington's disease. Philos. Trans. R. Soc. Pond. B. Biol. Sci. (1999) 354:1029–1034.
  • DIFIGLIA M, SAPP E, CHASE KO et al.: Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain. Science (1997) 277:1990–1993.
  • GUTEKUNST C, LI SH, YI H et al.: Nuclear and neuropil aggregates in Huntington's disease: relationship to neuropathology. J. Neurosci. (1999) 19:2522–2534.
  • KUEMMERLE S, GUTEKUNST CA, KLEIN AM et al.: Huntington aggregates may not predict neuronal death in Huntington's disease. Ann. NeuroL (1999) 46:842–849.
  • COOPER JK, SCHILLING G, PETERS MF et al.: Truncated N-terminal fragments of huntingtin with expanded glutamine repeats form nuclear and cytoplasmic aggregates in cell culture. Hum. MoL Genet. (1998) 7:783–790.
  • DAVIES SW, BEARDSALL K, TURMAINE M, DIFIGLIA M, ARONIN N, BATES GP: Are neuronal intranuclear inclusions the common neuropathology of triplet-repeat disorders with polyglutamine-repeat expansions? Lancet (1998) 351:131–133.
  • MEADE CA, DENG YP, FUSCO FR et al.: Cellular localization and development of neuronal intranuclear inclusions in striatal and cortical neurons in R6/2 transgenic mice. J. Comp. NeuroL (2002) 449:241–269.
  • ORDWAY JM, TALLAKSEN-GREENE S, GUTEKUNST CA et al.: Ectopically expressed GAG repeats cause intranuclear inclusions and a progressive late onset neurological phenotype in the mouse. Cell (1997) 91:753–763.
  • KLEMENT IA, SKINNER PJ, KAYTOR MD et al.: Ataxin-1 nuclear localization and aggregation: role in polyglutamine-induced disease in SCA1 transgenic mice. Cell (1998) 95:41–53.
  • KIM M, LEE HS, LAFORET G et al.: Mutant huntingtin expression in clonal striatal cells: dissociation of inclusion formation and neuronal survival by caspase inhibition. J. Neurosci. (1999) 19:964–973.
  • SAUDOU F, FINKBEINER S, DEVYS D, GREENBERG ME: Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusions. Cell (1998) 95: 55–66.
  • SISODIA SS: Nuclear inclusions in glutamine repeat disorders: are they pernicious, coincidental, or beneficial? Cell (1998) 95:1–4.
  • ARRASATE M, MITRA S, SCHWEITZER ES, SEGAL MR, FINKBEINER S: Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death. Nature (2004) 431:805–810.
  • DEDEOGLU A, KUBILUS JK, JEITNER TM et al.: Therapeutic effects of cystamine in a murine model of Huntington's disease. J. Neurosci. (2002) 22:8942–8950.
  • DEDEOGLU A, KUBILUS JK, YANG L et al.: Creatine therapy provides neuroprotection after onset of clinical symptoms in Huntington's disease transgenic mice. J. Neurochem. (2003) 85:1359–1367.
  • FERRANTE RJ, ANDREASSEN OA, JENKINS BG et al.: Neuroprotective effects of creatine in a transgenic mouse model of Huntington's disease. J. Neurosci. (2000) 20:4389–4397.
  • ANDREASSEN OA, DEDEOGLU A, FERRANTE RJ et al.: Creatine increase survival and delays motor symptoms in a transgenic animal model of Huntington's disease. NeurobioL Dis. (2001) 8:479–491.
  • FERRANTE RJ, ANDREASSEN OA, DEDEOGLU A et al.: Therapeutic effects of Coenzyme Q10 and remacemide in transgenic models of Huntington's disease. J. Neurosci. (2002) 22:1592–1599.
  • NUCIFORA FC Jr, SASAKI M, PETERS MF et al.: Interference by huntingtin and atrophin-1 with CBP-mediated transcription leading to cellular toxicity. Science (2001) 291:2423–2428.
  • BENCE NF, SAMPAT RM, KOPITO RR: Impairment of the ubiquitin-proteasome system by protein aggregation. Science (2001) 292:1552–1555.
  • PANOV AV, GUTEKUNST CA, LEAVITT BR et al.: Early mitochondrial calcium defects in Huntington's disease are a direct effect of polyglutamines. Nat. Neurosci. (2002) 5:731–736.
  • JIANG H, NUCIFORA FC, ROSS CA. DEFRANCO DB: Cell death triggered by polyglutamine-expanded huntingtin in a neuronal cell line is associated with degradation of CREB-binding protein. Hum. Mol. Genet. (2003) 12:1–12.
  • BEAL MF: Oxidative metabolism. Ann. NYAcad. Sci. (2000) 924:164–169.
  • DESAGHER S, MARTINOU JC: Mitochondria as the central control point of apoptosis. Trends Cell Biol. (2000) 10:369–377.
  • GREEN DR, REED JC: Mitochondria and apoptosis. Science (1998) 281: 1309–1312.
  • ROY S, NICHOLSON DW: Cross-talk in cell death signaling. J. Exp. Med. (2000) 192:21–26.
  • KIECHLE T, DEDEOGLU A, KUBILUS J et al.: Cytochrome C and caspase-9 expression in Huntington's disease. Neuromolecular Med. (2002) 1:183–195.
  • FERRANTE RJ, KUBILUS JK, LEE J et al.: Histone deacetylase inhibition by sodium butyrate chemotherapy ameliorates the neurodegenerative phenotype in Huntington's disease mice. J. Neurosci. (2003) 23:9418–9427.
  • FERRANTE RJ, RYU H, KUBILUS JK et al.: Chemotherapy for the brain: the antitumor antibiotic mithramycin prolongs survival in a mouse model of Huntington's disease. J. Neurosci. (2004) 24:10335–10342.
  • HOCKLY E, RICHON VM, WOODMAN B et al.: Suberoylanilide hydroxamic acid, a histone deacetylase inhibitor, ameliorates motor deficits in a mouse model of Huntington's disease. Proc. NatL Acad. Sci. USA (2003) 100:2041–2046.
  • GARDIAN G, BROWNE SE, CHOI DK et al.: Neuroprotective effects of phenylbutyrate in the N171-82Q transgenic mouse model of Huntington's disease. J. Biol. Chem. (2005) 280(1):556–563.
  • LORAND L, STEERING GE JR, LOWE-KRENTZ L: Formation of gamma-glutamyl-epsilon-lysine bridges between membrane proteins by a Ca2*-regulated enzyme in intact erythrocytes. J. SupramoL Struct. (1978) 9:427–440.
  • KAHLEM P, GREEN H, DJIAN P: Transglutaminase action imitates Huntington's disease: selective polymerization of huntingtin containing expanded polyglutamine. MoL Cell (1998) 1: 595–601.
  • IGARASHI S, KOIDE R, SHIMOHATA T et al.: Suppression of aggregate formation and apoptosis by transglutaminase inhibitors in cells expressing truncated DRPLA protein with an expanded polyglutamine stretch. Nat. Genet. (1998) 18:111-117. Expert Op/n. Emerging Drugs (2005) 10(2)
  • LESORT M, CHUN W, JOHNSON GVW, FERRANTE RJ: Tissue transglutaminase is increased in Huntington's disease brain. J. Neurochem. (1999) 73:2018–2027.
  • JEITNER TM, BOGDANOV MB, MATSON WR et al.: N-(y-L-glutamyfi-L-lysine is increased in cerebrospinal fluid of patients with Huntington's disease. J. Neurochem. (2001) 79:1109–1112.
  • REVESZ L, MODIG H: Cysteamine-induced increase of cellular glutathione-level: a new hypothesis of the radioprotective mechanism. Nature (1965) 207:430–431.
  • ROBITAILLE K, DAVIAU A, TUCHOLSKI J, JOHNSON GV, RANCOURT C, BLOUIN R: Tissue transglutaminase triggers oligomerization and activation of dual leucine zipper-bearing kinase in calphostin C-treated cells to facilitate apoptosis. Cell Death Differ. (2004) 11:542–549.
  • PALKOVITS M, BROWNSTEIN MJ, EIDEN LE et al.: Selective depletion of somatostatin in rat brain by cysteamine. Brain Res. (1982) 240:178–180.
  • MCDONNELL NB, DE GUZMAN RN, RICE WG, TURPIN JA, SUMMERS MF: Zinc ejection as a new rationale for the use of cystamine and related disulfide-containing antiviral agents in the treatment of AIDS. J. Med. Chem. (1997) 40: 1969–1976.
  • KARPUJ MV BECHER MW, SPRINGER JE et al.: Prolonged survival and decreased abnormal movements in transgenic model of Huntington's disease, with administration of the transglutaminase inhibitor cystamine. Nat. Med. (2002) 8:143–149.
  • FOX JH, BARBER DS, SINGH B et al.:Cystamine increases L-cysteine levels in Huntington's disease transgenic mouse brain and in a PC12 model of polyglutamine aggregation. J. Neurochem. (2004) 91:413–422.
  • BAILEY CD, JOHNSON GV: Tissue transglutaminase contributes to disease progression in the R6/2 Huntington's disease mouse model via aggregate-independent mechanisms. J. Neurochem. (2005) 92:83–92.
  • PRESCOTT LF, NEWTON RW, SWAINSON CP, WRIGHT N, FORREST AR, MATTHEW H: Successful treatment of severe paracetamol overdosage with cysteamine. Lancet (1974) 1:588–592.
  • GM-IL WA, THOENE JG, SCHNEIDER JA: Cystinosis. N Engl Med. (2002) 347(2):111–121.
  • CORDEN BJ, SCHULMAN JD, SCHNEIDER JA, THOENE JG: Adverse reactions to oral cysteamine use in nephropathic cystinosis. Dev. Pharmacol Ther. (1981) 3:25–30.
  • FRIEDLANDER RM: Apoptosis and caspases in neurodegenerative diseases. N Engl. J. Med. (2003) 348:1365–1375.
  • YRJANHEIKKI J, KEINANEN R, PELLIKKA M, HOKFELT T, KOISTINAHO J: Tetracyclines inhibit microglial activation and are neuroprotective in global brain ischemia. Proc. Nati Acad. Sci. USA (1998) 95:15769–15774.
  • CHEN M, ONA VO, LI M et al.: Minocycline inhibits caspase-1 and caspase-3 expression and delays mortality in a transgenic mouse model of Huntington's disease. Nat. Med. (2000) 6:797–801.
  • SANCHEZ MEJIA RO, ONA VO, LI M, FRIEDLANDER RM: Minocycline reduces traumatic brain injury-mediated caspase-1 activation, tissue damage, and neurological dysfunction. Neurosurgery (2001) 48:1393–1401.
  • TIKKA T, FIEBICH BL, GOLDSTEINS G, KEINANEN R, KOISTINAHO J: Minocycline, a tetracycline derivative, is neuroprotective against excitotoxicity by inhibiting activation and proliferation of microglia. Neurosci. (2001) 21:2580–2588.
  • WU DC, JACKSON-LEWIS V, VILA M et al.: Blockade of microglial activation is neuroprotective in the 1-methyl-4-phenyl-1,2,3,6- tetrahydropyridine mouse model of Parkinson's disease. J. Neurosci. (2002) 22:1763–1771.
  • DU Y, MA Z, LIN S et al.: Minocycline prevents nigrostriatal dopaminergic neurodegeneration in the MPTP model of Parkinson's disease. Proc. Nati Acad. Sci. USA (2001) 98:14669–14674.
  • ZHU S, STAVROVSKAYA IG, DROZDA M et al.: Minocycline inhibits cytochrome c release and delays progression of ALS in mice. Nature (2002) 417:72–78.
  • WANG X, ZHU S, DROZDA M et al.: Minocycline inhibits caspase-independent and -dependent mitochondrial cell death pathways in models of Huntington's disease. Proc. Natl Acad. Sci. USA (2003) 100:10483–10487.
  • SCHERZINGER E, LURZ R, TURMAINE M et al.: Huntingtin-encoded polyglutamine expansions form amyloid-like protein aggregates in vitro and in vivo. Cell (1997) 90(3):549–558.
  • SMITH DL, WOODMAN B, MAHAL A et al.: Minocycline and doxycycline are not beneficial in a model of Huntington's disease. Ann. NeuroL (2003) 54:186–196.
  • HERSCH S, FINK K, VONSATTEL JP, FRIEDLANDER RM: Minocycline is protective in a mouse model of Huntington's disease. Ann. Neurol (2003) 54:841; author reply 842–843.
  • BANTUBUNGI K, JACQUARD C, GRECO A et al.: Minocycline in phenotypic models of Huntington's disease. Neurobiol Dis. (2005) 18:206–217.
  • GONI-ALLO B, RAMOS M, JORDAN J, AGUIRRE N: In vivo studies on the protective role of minocycline against excitotoxicity caused by malonate or N-methyl-D-aspartate. Exp. Neurol (2005) 191:326–330.
  • CARRERAS I, KUBILUS JK, SMITH K et al.: Combination therapy using minocycline and coenzyme Q10 in R6/2 transgenic HD mice. Program No. 208.10. Abstract Viewer/Itinerary Planner. Society for Neuroscience, Washington, DC (2003).
  • HUNTINGTON STUDY GROUP: Minocycline safety and tolerability in Huntington's disease. Neurology (2004) 63:547–549.
  • THOMAS M, ASHIZAWA T, JANKOVIC J: Minocycline in Huntington's disease: a pilot study. Mov. Disord. (2004) 19:692–695.
  • BONELLI RM, HODL AK, HOFMANN P, KAPFHAMMER HP: Neuroprotection in Huntington's disease: a 2-year study on minocycline. Int. Clin. Psychopharmacol (2004) 19:337–342.
  • DENOVAN-WRIGHT EM, DEVARAJAN S, DURSUN SM, ROBERTSON HA: Maintained improvement with minocycline of a patient with advanced Huntington's disease. Psychopharmacol (2002) 16:393–394.
  • BLUM D, CHTARTO A, TENENBAUM L, BROTCHI J, LEVI VIER M: Clinical potential of minocycline for neurodegenerative disorders. NeurobioL Dis. (2004) 17:359–366.
  • YONG VW, WELLS J, GIULIANI F, CASHA S, POWER C, METZ LM: The promise of minocycline in neurology Lancet NeuroL (2004) 3:744–751.
  • TAN HH: Antibacterial therapy for acne: a guide to selection and use of systemic agents. Am. J. Clin. DermatoL (2003) 4:307–314.
  • EMERY P, SUAREZ-ALMAZOR M: Rheumatoid arthritis. Clin. Evid. (2003) 9:1349–1371.
  • KNOWLES SR, SHAPIRO L, SHEAR NH: Serious adverse reactions induced by minocycline. Report of 13 patients and review of the literature. Arch. Dermatol. (1996) 132:934–939.
  • STEFFAN JS, BODAI L, PALLOS J et al.: Histone deacetylase inhibitors arrest polyglutamine-dependent neurodegeneration in Drosophila. Nature (2001) 413:739–743.
  • HAKE SB, MAO A, ALLIS CD: Linking the epigenetic 'language' of covalent histone modifications to cancer. Br. J. Cancer (2004) 90:761–769.
  • BLANCO G, FU H, MENDEZ C, KHOSLA C, SALAS JA: Deciphering the biosynthetic origin of the aglycone of the aureolic acid group of anti-tumor agents. Chem. Biol. (1996) 3:193–196.
  • RALSTON SH: Pathogenesis and management of cancer associated hypercalcaemia. Cancer Surv. (1994) 21:179–196.
  • CHAKRABARTI S, BHATTACHARYYA D, DASGUPTA D: Structural basis of DNA recognition by anticancer antibiotics, chromomycin A3, and mithramycin: roles of minor groove width and ligand flexibility. Biopolymers (2001) 56:85–95.
  • HESCOCK H Jr, PARKER M, WANG TY, BALLINGER R, BALDUCCI L: Metastatic carcinoma of unknown primary: complete response to second-line treatment with plicamycin. Am. J. Med. Sci. (1989) 298:34–37.
  • PRADO L, LOMBO F, BRANA AF, MENDEZ C, ROHR J, SALAS JA: Analysis of two chromosomal region adjacent to genes for a Type II polyketide synthase involved in the biosynthesis of the antitumor polyketide mithramycin in Streptomyces argillaceus. Mol. Gen. Genet. (1999) 261:216–225.
  • RYAN WG: Treatment of Paget's disease of bone with mithramycin. Clin. Orthop. (1977) 127:106–110.
  • KENNEDY BJ: Mithramycin therapy in testicular cancer. J. Urol. (1972) 107:429–432.
  • RANSOHOFF J, MARTIN BF, MEDREK TJ, HARRIS MN, GOLOMB FM, WRIGHT JC: Preliminary clinical study of mithramycin (nsc-24559) in primary tumors of the central nervous system. Cancer Chemother. Rep. (1965) 49:51–57.
  • HAGEN G, DENNIG J, PREISS A, BEATO M, SUSKE G: Functional analyses of the transcription factor Sp4 reveal properties distinct from Spl and Sp3. J. Biol. Chem. (1995) 270:24989–24994.
  • MAJELLO B, DE LUCA P, SUSKE G, LANIA L: Differential transcriptional regulation of c-myc promoter through the same DNA binding sites targeted by Spl-like proteins. Oncogene (1995) 10:1841–1848.
  • CHATTERJEE S, ZAMAN K, RYU H, CONFORTO A, RATAN RR: Sequence-selective DNA binding drugs mithramycin A and chromomycin A3 are potent inhibitors of neuronal apoptosis induced by oxidative stress and DNA damage in cortical neurons. Ann. NeuroL (2001) 49:345–354.
  • DUNAH AW, JEONG H, GRIFFIN A et al.: Sp 1 and TAFII130 transcriptional activity disrupted in early Huntington's disease. Science (2002) 296:2238–2243.
  • LI SH, CHENG AL, ZHOU H et al.: Interaction of Huntington's disease protein with transcriptional activator Spl. MoL Cell Biol. (2002) 22:1277–1287.
  • SISSI C, MORO S, RICHTER S et al.: DNA-interactive anticancer aza-anthrapyrazoles: biophysical and biochemical studies relevant to the mechanism of action. MoL Pharmacol. (2001) 59:96–103.
  • MARKS PA, RICHON VM, RIFKIND RA. Nucleotide, protein histone deacetylase inhibitors: inducers of differentiation or apoptosis of transformed cells. J. Need Cancer Inst. (2000) 92:1210–1216.
  • CONLEY BA, EGORIN MJ, TAIT N et al.: Phase I study of the orally administered butyrate prodrug, tributyrin, in patients with solid tumors. Clin. Cancer Res. (1998) 4:629–634.
  • SHARMA SK, MICHAELIS C, LEE KY, WANG JH, WEEKS G: Binding and catalytic properties of the Cdc2 and Crp proteins of Dictyostelium. Eur. j Biochem. (1999) 260:603–608.
  • RICHON VM, ZHOU X, RIFKIND RA, MARKS Pk Histone deacetylase inhibitors: development of suberoylanilide hydroxamic acid (SAHA) for the treatment of cancers. Blood Cells MoL Dis. (2001) 27:260–264.
  • GORIN NC, ESTEY E, JONES RJ, LEVITSKY HI, BORRELLO I, SLAVIN S: New developments in the therapy of acute myelocytic leukemia. Hematology (Am. Soc. HematoL Educ. Program) (2000) 179:69–89.
  • CHANG JG, HSIEH-LI HM, JONG YJ et al.: Treatment of spinal muscular atrophy by sodium butyrate. Proc. Nail. Acad. Sci. USA (2001) 98:9808–9813.
  • EGORIN MJ, YUAN ZM, SENTZ DL, PLAISANCE K, EISEMAN JL: Plasma pharmacokinetics of butyrate after intravenous administration of sodium butyrate or oral administration of tributyrin or sodium butyrate to mice and rats. Cancer Chemother. PharmacoL (1999) 43:445–453.
  • SEALY L, CHALKLEY R: The effect of sodium butyrate on histone modification. Cell (1978) 14: 115–121.
  • CANDIDO EP, REEVES R, DAVIE JR: Sodium butyrate inhibits histone deacetylation in cultured cells. Cell (1978) 14:105–113.
  • COLLINS AF, PEARSON HA, GIARDINA P, MCDONAGH KT, BRUSILOW SW, DOVER GJ: Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial. Blood (1995) 85:43–49.
  • SHER GD, GINDER GD, LITTLE J, YANG S, DOVER GJ, OLIVIERI NF: Extended therapy with intravenous arginine butyrate in patients with beta-hemoglobinopathies. New EngL J. Med. (1995) 332:1606–1610.
  • BATSHAW ML, MACARTHUR RB, TUCHMAN M: Alternative pathway therapy for urea cycle disorders: twenty years later. J. Pediatr. (2001) 138:S46–S55.
  • BERGS, SERABE B, ALEKSIC A et al.: Pharmacokinetics and cerebrospinal fluid penetration of phenylacetate and phenylbutyrate in the nonhuman primate. Cancer Chemother. PharmacoL (2001) 47:385–390.
  • BURLINA AB, OGIER H, KORALL H, TREFZ FK: Long-term treatment with Expert Op/n. Emerging Drugs (2005) 10(2) sodium phenylbutyrate in ornithine transcarbamylase-deficient patients. Mol. Genet. Metab. (2001) 72:351–355.
  • GILBERT J, BAKER SD, BOWLING MK et al.: A Phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies. Clin. Cancer Res. (2001) 7:2292–2300.
  • SAMID D: Therapeutic targeting of transcription in acute promyelocytic leukemia by use of an inhibitor of histone deacetylase. J. Natl. Cancer Inst. (1999) 91:475–476.
  • MARKS PA, RICHON VM, KELLY WK, CHIAO JH, MILLER T: Histone deacetylase inhibitors: development as cancer therapy. Novartis Found. Symp. (2004) 259:269–281.
  • MCGUINNESS MC, ZHANG HP, SMITH KD: Evaluation of pharmacological induction of fatty acid beta-oxidation in X-linked adrenoleukodystrophy. MoL Genet. Metab. (2001) 74:256–263
  • WEI H, KEMP S, MCGUINNESS MC, MOSER AB, SMITH KD: Pharmacological induction of peroxisomes in peroxisome biogenesis disorders. Ann. NeuroL (2000) 47:286–296.
  • KEMP S, WEI HM, LU JF et al.: Gene redundancy and pharmacological gene therapy: implications for X-linked adrenoleukodystrophy. Nat. Med. (1998) 4:1261–1268.
  • DOVER GJ, BRUSILOW S, CHARACHE S: Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate. Blood (1994) 84:339–343.
  • DOVER GJ: Hemoglobin switching protocols in thalassemia. Experience with sodium phenylbutyrate and hydroxyurea. Ann. IVY Acad. Sci. (1998) 850:80–86.
  • BROUILLET E, HANTRAYE P, FERRANTE RJ et al.: Chronic mitochondrial energy impairment produces selective striatal degeneration and abnormal choreiform movements in primates. Proc. Nail. Acad. Sci. USA (1995) 92:7105–7109.
  • BROWNE SE, BOWLING AC, MACGARVEY U et al.: Oxidative damage and metabolic dysfunction in Huntington's disease: selective vulnerability of the basal ganglia. Ann. NeuroL (1997) 41:646–653.
  • GUM, GASH MT, MANN VM, JAVOY-AGID F, COOPER JM, SCHAPIRA Al-TV: Mitochondrial defect in Huntington's disease caudate nucleus. Ann. NeuroL (1996) 39:385–389.
  • JENKINS B, KOROSHETZ W, BEAL MF, ROSEN B: Evidence for an energy metabolism defect in Huntington's disease using localized proton spectroscopy. Neurology (1993) 43:2689–2695.
  • KOROSHETZ WJ, JENKINS BG, ROSEN BR, BEAL MF: Energy metabolism defects in Huntington's disease and possible therapy with coenzyme Q10. Ann. NeuroL (1997) 41: 160–165.
  • BESSMAN SP, GEIGER PJ: Transport of energy in muscle: the phosphorylcreatine shuttle. Science (1981) 211:448–452.
  • MATTHEWS RT, YANG L, JENKINS BG et al.: Neuroprotective effects of creatine and cyclocreatine in animal models of Huntington's disease. Neurosci. (1998) 18: 156–163.
  • CARTER AJ, MULLER RE, PSCHORN U, STRANSKY W: Preincubation with creatine enhances levels of creatine phosphate and prevents anoxic damage in rat hippocampal slices. Neurochem. (1995) 64:2691–2699.
  • MATTHEWS RT, FERRANTE RJ, KLIVENYI P et al.: Creatine and cyclocreatine attenuate MPTP neurotoxicity. Exp. NeuroL (1999) 157:142–149.
  • KLIVENYI P, FERRANTE RJ, MATTHEWS RT et al.: Neuroprotective effects of creatine in a transgenic animal model of ALS. Nat. Med. (1999) 5:347–350.
  • VERBESSEM P, LEMIERE J, EIJNDE B et al.: Creatine supplementation in Huntington's disease: a placebo-controlled pilot trial. Neurology (2003) 61:925–930.
  • TABRIZI SJ, BLAMIRE AM, MANNERS DN et al.: Creatine therapy for Huntington's disease: clinical and MRS findings in a 1-year pilot study. Neurology (2003) 61:141–142.
  • BENDER A, AUER DP, MERL T et al.: Creatine supplementation lowers brain glutamate levels in Huntington's disease. Neurol. (2005) 252:36–41.
  • LAWLER JM, BARNES WS, WU G, SONG W, DEMAREE S: Direct antioxidant properties of creatine. Biochem. Biophys. Res. Comm. (2002) 290:47–52.
  • BENZI G: Is there a rationale for the use of creatine either as nutritional supplementation or drug administration in humans participating in sport? PharmacoL Res. (2000) 41:255–264.
  • COOKE WH, GRANDJEAN PW, BARNES WS. Effect of oral creatine supplementation on power output and fatigue during bicycle ergometry. J. AppL Physiol. (1995) 78:670–673.
  • TARNOPOLSKY MA, BEAL MF: Potential for creatine and other therapies targeting cellular energy dysfunction in neurological disorders. Ann. NeuroL (2001) 49:561–574.
  • WYSS M, SCHULZE A: Health implications of creatine: can oral creatine supplementation protect against neurological and atherosclerotic disease? Neurosci. (2002) 112:243–260.
  • MERTSCHENK B, GLOXHUBER C, WALLIMANN T: Health assessment of creatine as a dietary supplement. Dtsch. Lebensm. Rundsch. (2001) 97:250–257.
  • BEYER RE: An analysis of the role of coenzyme Q in free radical generation and as an antioxidant. Biochem. Cell Biol. (1992) 70:390–403.
  • NOACK H, KUBE U, AUGUSTIN W: Relations between tocopherol depletion and coenzyme Q during lipid peroxidation in rat liver mitochondria. Free. Radic. Res. (1994) 20:375–386.
  • KAGAN V, SERBINOVA E, PACKER L: Antioxidant effects of ubiquinones in microsomes and mitochondria are mediated by tocopherol recycling. Biochem. Biophys. Res. Commun. (1990) 169:851–857.
  • ECHTAY KS, ROUSSEL D, ST-PIERRE J et al.: Superoxide activates mitochondrial uncoupling proteins. Nature (2002) 415:96–99.
  • CASTEILLA L, RIGOULET M, PENICAUD L: Mitochondrial ROS metabolism: modulation by uncoupling proteins. IUBMB Life (2001) 52:181–188.
  • NISHIKAWA Y, TAKAHASHI M, YORIFUJI S et al.: Long-term coenzyme Q„ therapy for a mitochondrial encephalomyopathy with cytochrome c oxidase deficiency: A31P NMR study. Neurology (1989) 39:399–403.
  • IHARA Y, NAMBA R, KURODA S, SATO T, SHIRABE T: Mitochondrial encephalomyopathy (MELAS): pathological study and successful therapy with coenzyme Q„ and idebenone. J. NeuroL Sci. (1989) 90:263–271.
  • CHARIOT P, BRUGIERES P, ELIEZER-VANEROT MC, GENY C, BINAGHI M, CESARO P: Choreic movements and MRI abnormalities in the subthalamic nuclei reversible after administration of coenzyme Q„ and multiple vitamins in a patient with bilateral optic neuropathy. Mov. Disord. (1999) 14:855–859.
  • ABE K, FUJIMURA H, NISHIKAWA Y et al.: Marked reduction in CSF lactate and pyruvate levels after CoQ therapy in a patient with mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes (MELAS). Acta Neurol. Scand. (1991) 83:356–359.
  • TOMASETTI M, LITTARRU GP, STOCKER R, ALLEVA R: Coenzyme Q10 enrichment decreases oxidative DNA damage in human lymphocytes. Free Radic. Biol. Med. (1999) 27:1027–1032.
  • CHAN A, REICHMANN H, KOGEL A et al.: Metabolic changes in patients with mitochondrial myopathies and effects of coenzyme Q„ therapy. J. Neurol. (1998) 245:681–685.
  • SCHILLING G, SAVONENKO AV, COONFIELD ML et al.: Environmental, pharmacological, and genetic modulation of the HD phenotype in transgenic mice. Exp. Neurol. (2004) 187:137–149.
  • BEAL MF, HENSHAW R, JENKINS BG, ROSEN BR, SCHULZ JB: Coenzyme Q10 and nicotinamide block striatal lesions produced by the mitochondrial toxin malonate. Ann. Neurol. (1994) 36:882–888.
  • LASS A, FORSTER MJ, SOHAL RS: Effects of coenzyme Q„ and a-tocopherol administration on their tissue levels in the mouse: elevation of mitochondrial alpha tocopherol by coenzyme Q. Free Radic. Biol. Med. (1999) 26:1375–1382.
  • SHULTS CW, OAKES D, KIEBURTZ K et al.: Effects of coenzyme Q10 in early Parkinson's disease: evidence of slowing of the functional decline. Arch Neurol. (2002) 59:1541–1550.
  • SMITH K, MATSON S, CORMIER K et al.: Therapeutic effects of high dose administration of coenzyme Q10 in the R6/2 model of Huntington's disease. Abstract Viewer/Itinerary Planner. Society for Neuroscience, Washington, DC, USA (2004).
  • OKAMOTO T, FUKUI K, NAKAMOTO M et al.: Serum levels of coenzyme Q10 and lipids in patients during total parenteral nutrition. J. Num. Sci. VitaminoL (1986) 32: 1–12.
  • MOLYNEUX S, FLORKOWSKI C, LEVER M, GEORGE P: The bioavailability of coenzyme Q10 supplements available in New Zealand differs markedly. NZ Med. J. (2004) 117(1203):U1108.
  • WEBER C, SEJERSGARD JAKOBSEN T, MORTENSEN SA, PAULSEN G, HOLMER G: Antioxidative effect of dietary coenzyme Q_„ in human blood plasma. Int. J. Vitam. Num. Res. (1994) 64:311–315.
  • MATTHEWS RT, YANG L, BROWNE S, BAIK M, BEAL MF: Coenzyme Q„ administration increases brain mitochondrial concentrations and exerts neuroprotective effects. Proc. Nail. Acad. Sci. USA (1998) 95:8892–8897.
  • HUNTINGTON STUDY GROUP: A randomized, placebo-controlled trial of coenzyme Q10 and remacemide in Huntington's disease. Neurology (2001) 57:397–404.
  • MUSUMECI O, NAINI A, SLONIM AE et al.: Familial cerebellar ataxia with muscle coenzyme Q10 deficiency. Neurology (2001) 56:849–855.

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