Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteersSusan P Perrine
HemaQuest Pharmaceuticals, Inc, Boston, MA 02110, USA
J Clin Pharmacol 51:1186-94. 2011
..The favorable pharmacokinetics (PK) profiles and safety findings indicate that SDMB warrants further investigation for treatment of anemic subjects with beta hemoglobinopathies...
Histone deacetylase inhibitors are potent inducers of gene expression in latent EBV and sensitize lymphoma cells to nucleoside antiviral agentsSajal K Ghosh
Cancer Center, Boston University School of Medicine, 72 East Concord Street, Boston, MA 02118, USA
Blood 119:1008-17. 2012
..The effectiveness and potency of these HDAC inhibitors make them potentially applicable as sensitizers to antivirals for the treatment of EBV-associated lymphomas...
Erythroid Kruppel-like factor (EKLF) is recruited to the gamma-globin gene promoter as a co-activator and is required for gamma-globin gene induction by short-chain fatty acid derivativesSusan P Perrine
Cancer Center, Boston University School of Medicine, Boston, MA 02118, USA
Eur J Haematol 82:466-76. 2009
..We sought to determine if EKLF is also required for activation of the gamma-globin gene by short-chain fatty acid (SCFA) derivatives, which are now entering clinical trials...
Short-chain fatty acids induce gamma-globin gene expression by displacement of a HDAC3-NCoR repressor complexRishikesh Mankidy
Gene Regulation Laboratories, Inc, Newton, MA, USA
Blood 108:3179-86. 2006
..These studies demonstrate new dynamic alterations in transcriptional regulatory complexes associated with SCFAD-induced activation of the gamma-globin gene and provide a specific molecular target for potential therapeutic intervention...
Fetal globin gene inducers: novel agents and new potentialSusan P Perrine
Cancer Center and Hemoglobinopathy Thalassemia Research Unit, Department of Medicine, Pediatrics, Pharmacology and Experimental Therapeutics, Boston University School of Medicine, Boston, Massachusetts, USA
Ann N Y Acad Sci 1202:158-64. 2010
..These refinements offer renewed potential to apply fetal globin induction as a treatment approach in patient-friendly regimens that can be used worldwide...
Induction of fetal globin in beta-thalassemia: Cellular obstacles and molecular progressSusan P Perrine
Hemoglobinopathy Thalassemia Research Unit, Boston University School of Medicine, 80 E Concord St, L 908, Boston, MA 02118, USA
Ann N Y Acad Sci 1054:257-65. 2005
..Both approaches now offer excellent potential for tolerable, definitive treatment of beta-thalassemia...
A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcersLillian McMahon
Cancer Center and Hemoglobinopathy Thalassemia Research Unit, Boston University School of Medicine, Boston, MA 02118, USA
Br J Haematol 151:516-24. 2010
..These findings strongly suggest that Arginine Butyrate merits further evaluation for the treatment of refractory sickle cell leg ulcers in larger trials...
Identification of novel small-molecule inducers of fetal hemoglobin using pharmacophore and 'PSEUDO' receptor modelsRegine Bohacek
Boston De Novo Design, 50 Commonwealth Ave, Boston, MA 02116, USA
Chem Biol Drug Des 67:318-28. 2006
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Short, discontinuous exposure to butyrate effectively sensitizes latently EBV-infected lymphoma cells to nucleoside analogue antiviral agentsSajal K Ghosh
Cancer Research Center, Boston University School of Medicine, 715 Albany Street, K701, Boston, MA 02118, USA
Blood Cells Mol Dis 38:57-65. 2007
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Histone deacetylase inhibitors reverse CpG methylation by regulating DNMT1 through ERK signalingSibaji Sarkar
Cancer Center, Boston University School of Medicine, Boston, MA 02118, USA
Anticancer Res 31:2723-32. 2011
..These results demonstrate that HDACs regulate gene methylation, in addition to the established and reciprocal ability of CpG methylation to recruit HDACs to repress transcription...
Fetal globin stimulant therapies in the beta-hemoglobinopathies: principles and current potentialSusan P Perrine
Hemoglobinopathy Thalassemia Research Unit, Boston University School of Medicine, Boston, Massachusetts, USA
Pediatr Ann 37:339-46. 2008
..Development of this therapeutic avenue will require close collaboration among treating and academic physicians, families and patients, funding agencies, and researchers...
Enhancement of growth and survival and alterations in Bcl-family proteins in beta-thalassemic erythroid progenitors by novel short-chain fatty acid derivativesSerguei Castaneda
Hemoglobinopathy-Thalassemia Research Unit and Cancer Research Center, Boston University School of Medicine, 715 Albany Street, L-908, Boston, MA 02118, USA
Blood Cells Mol Dis 35:217-26. 2005
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Advances in Virus-Directed Therapeutics against Epstein-Barr Virus-Associated MalignanciesSajal K Ghosh
Cancer Center, Boston University School of Medicine, Boston, MA 02118, USA
Adv Virol 2012:509296. 2012
..In this paper we provide a brief account of new virus-targeted therapeutic approaches against EBV-associated malignancies...
A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus-associated lymphoid malignanciesSusan P Perrine
Cancer Research Center, Boston University School of Medicine, Boston, MA 02118, USA
Blood 109:2571-8. 2007
..The combination of arginine butyrate and ganciclovir was reasonably well-tolerated and appears to have significant biologic activity in vivo in EBV(+) lymphoid malignancies which are refractory to other regimens...
Alpha-lipoic acid induces p27Kip-dependent cell cycle arrest in non-transformed cell lines and apoptosis in tumor cell linesKaryn van de Mark
Cancer Research Center, Boston University School of Medicine, Boston, Massachusetts 02118, USA
J Cell Physiol 194:325-40. 2003
..The differential selectivity of the pro-apoptotic effects of alpha-lipoic acid for transformed cells supports its potential use in the treatment of neoplastic disorders...