Publication List

Mechanism of Action of 2-Aminobenzamide HDAC Inhibitors in Reversing Gene Silencing in Friedreich's Ataxia. Soragni E, Chou CJ, Rusche JR, Gottesfeld JM. Front Neurol. 2015 Mar 5;6:44. doi: 10.3389/fneur.2015.00044. eCollection 2015. PMID: 25798128

RNA toxicity and missplicing in the common eye disease fuchs endothelial corneal dystrophy. Du J, Aleff RA, Soragni E, Kalari K, Nie J, Tang X, Davila J, Kocher JP, Patel SV, Gottesfeld JM, Baratz KH, Wieben ED.  J Biol Chem. 2015 Mar 6;290(10):5979-90. doi: 10.1074/jbc.M114.621607. Epub 2015 Jan 15. PMID:25593321

Epigenetic therapy for Friedreich ataxia. Soragni E, Miao W, Iudicello M, Jacoby D, De Mercanti S, Clerico M, Longo F, Piga A, Ku S, Campau E, Du J, Penalver P, Rai M, Madara JC, Nazor K, O'Connor M, Maximov A, Loring JF, Pandolfo M, Durelli L, Gottesfeld JM, Rusche JR.  Ann Neurol. 2014 Oct;76(4):489-508. doi: 10.1002/ana.24260. Epub 2014 Sep 16.  PMID:25159818

Quantitative proteomic analysis identifies targets and pathways of a 2-aminobenzamide HDAC inhibitor in Friedreich's ataxia patient iPSC-derived neural stem cells. Shan B, Xu C, Zhang Y, Xu T, Gottesfeld JM, Yates JR 3rd.  J Proteome Res. 2014 Nov 7;13(11):4558-66. doi: 10.1021/pr500514r. Epub 2014 Jun 26.  PMID:24933366

Introduction to thematic minireview series: Development of human therapeutics based on induced pluripotent stem cell (iPSC) technology.  Rao M, Gottesfeld JM.  J Biol Chem. 2014 Feb 21;289(8):4553-4. doi: 10.1074/jbc.R113.543652. Epub 2013 Dec 20.  PMID:24362035

Length-dependent CTG·CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells. Du J, Campau E, Soragni E, Jespersen C, Gottesfeld JM.  Hum Mol Genet. 2013 Dec 20;22(25):5276-87. doi: 10.1093/hmg/ddt386. Epub 2013 Aug 9.  PMID: 23933738

Increasing frataxin gene expression with histone deacetylase inhibitors as a therapeutic approach for Friedreich's ataxia. Gottesfeld J, Rusche JR, Pandolfo M.  J Neurochem. 2013 Aug;126 Suppl 1:147-54. doi: 10.1111/jnc.12302. Review.  PMID:23859350

Minireview series on sirtuins: from biochemistry to health and disease. Denu J, Gottesfeld JM.  J Biol Chem. 2012 Dec 14;287(51):42417-8. doi: 10.1074/jbc.R112.428862. Epub 2012 Oct 18. No abstract available.  PMID:23086946

Role of mismatch repair enzymes in GAA·TTC triplet-repeat expansion in Friedreich ataxia induced pluripotent stem cells. Du J, Campau E, Soragni E, Ku S, Puckett JW, Dervan PB, Gottesfeld JM.  J Biol Chem. 2012 Aug 24;287(35):29861-72. doi: 10.1074/jbc.M112.391961. Epub 2012 Jul 13.  PMID: 22798143

Rationale for the development of 2-aminobenzamide histone deacetylase inhibitors as therapeutics for Friedreich ataxia. Soragni E, Xu C, Plasterer HL, Jacques V, Rusche JR, Gottesfeld JM.  J Child Neurol. 2012 Sep;27(9):1164-73. doi: 10.1177/0883073812448533. Epub 2012 Jul 4. Review.  PMID: 22764181

Chromatin structure determines accessibility of a hairpin polyamide-chlorambucil conjugate at histone H4 genes in pancreatic cancer cells. Jespersen C, Soragni E, James Chou C, Arora PS, Dervan PB, Gottesfeld JM.  Bioorg Med Chem Lett. 2012 Jun 15;22(12):4068-71. doi: 10.1016/j.bmcl.2012.04.090. Epub 2012 Apr 25.  PMID: 22607671

Histone deacetylase (HDAC) inhibitors targeting HDAC3 and HDAC1 ameliorate polyglutamine-elicited phenotypes in model systems of Huntington's disease. Jia H, Pallos J, Jacques V, Lau A, Tang B, Cooper A, Syed A, Purcell J, Chen Y, Sharma S, Sangrey GR, Darnell SB, Plasterer H, Sadri-Vakili G, Gottesfeld JM, Thompson LM, Rusche JR, Marsh JL, Thomas EA.  Neurobiol Dis. 2012 May;46(2):351-61.  PMID: 22590724

A gene expression phenotype in lymphocytes from Friedreich ataxia patients. Coppola G, Burnett R, Perlman S, Versano R, Gao F, Plasterer H, Rai M, Saccá F, Filla A, Lynch DR, Rusche JR, Gottesfeld JM, Pandolfo M, Geschwind DH.  Ann Neurol. 2011 Nov;70(5):790-804. doi: 10.1002/ana.22526.  PMID: 22162061

Evaluation of histone deacetylase inhibitors as therapeutics for neurodegenerative diseases. Soragni E, Xu C, Cooper A, Plasterer HL, Rusche JR, Gottesfeld JM. Methods Mol Biol. 2011;793:495-508. doi: 10.1007/978-1-61779-328-8_32. PMID: 21913121

Introduction to the thematic minireview series on epigenetics. Gottesfeld JM.  J Biol Chem. 2011 May 27;286(21):18345-6. doi: 10.1074/jbc.R111.243527. Epub 2011 Mar 24. No abstract available.  PMID: 21454488

Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model. Sandi C, Pinto RM, Al-Mahdawi S, Ezzatizadeh V, Barnes G, Jones S, Rusche JR, Gottesfeld JM, Pook MA.  Neurobiol Dis. 2011 Jun;42(3):496-505. doi: 10.1016/j.nbd.2011.02.016. Epub 2011 Mar 17.  PMID: 21397024

Friedreich's ataxia induced pluripotent stem cells model intergenerational GAA⋅TTC triplet repeat instability. Ku S, Soragni E, Campau E, Thomas EA, Altun G, Laurent LC, Loring JF, Napierala M, Gottesfeld JM. Cell Stem Cell. 2010 Nov 5;7(5):631-7. doi: 10.1016/j.stem.2010.09.014. PMID: 21040903

Chemical biology meets biological chemistry minireview series. Cravatt BF, Gottesfeld JM. J Biol Chem. 2010 Apr 9;285(15):11031-2. doi: 10.1074/jbc.R110.113126. Epub 2010 Feb 10. No abstract available.  PMID: 20147295

Two new pimelic diphenylamide HDAC inhibitors induce sustained frataxin upregulation in cells from Friedreich's ataxia patients and in a mouse model. Rai M, Soragni E, Chou CJ, Barnes G, Jones S, Rusche JR, Gottesfeld JM, Pandolfo M. PLoS One. 2010 Jan 21;5(1):e8825. doi: 10.1371/journal.pone.0008825. PMID:20098685

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis. Hutt DM, Herman D, Rodrigues AP, Noel S, Pilewski JM, Matteson J, Hoch B, Kellner W, Kelly JW, Schmidt A, Thomas PJ, Matsumura Y, Skach WR, Gentzsch M, Riordan JR, Sorscher EJ, Okiyoneda T, Yates JR 3rd, Lukacs GL, Frizzell RA, Manning G, Gottesfeld JM, Balch WE.  Nat Chem Biol. 2010 Jan;6(1):25-33. doi: 10.1038/nchembio.275. Epub 2009 Dec 6.  PMID: 19966789

Potent activity against K562 cells by polyamide-seco-CBI conjugates targeting histone H4 genes. Minoshima M, Chou JC, Lefebvre S, Bando T, Shinohara K, Gottesfeld JM, Sugiyama H. Bioorg Med Chem. 2010 Jan 1;18(1):168-74. doi: 10.1016/j.bmc.2009.11.005. Epub 2009 Nov 11. PMID: 19962902