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Molecular Mechanisms and Therapeutics for the GAA·TTC Expansion Disease Friedreich Ataxia

Neurotherapeutics, 2019-10, Vol.16 (4), p.1032-1049

Gottesfeld, Joel M.

2019

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The Effects of Pharmacological Inhibition of Histone Deacetylase 3 (HDAC3) in Huntington's Disease Mice

PloS one, 2016-03, Vol.11 (3), p.e0152498

2016

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Pimelic Diphenylamide 106 Is a Slow, Tight-binding Inhibitor of Class I Histone Deacetylases

The Journal of biological chemistry, 2008-12, Vol.283 (51), p.35402-35409

2008

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Two new pimelic diphenylamide HDAC inhibitors induce sustained frataxin upregulation in cells from Friedreich's ataxia patients and in a mouse model

PloS one, 2010-01, Vol.5 (1), p.e8825

2010

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RNA Toxicity and Missplicing in the Common Eye Disease Fuchs Endothelial Corneal Dystrophy

The Journal of biological chemistry, 2015-03, Vol.290 (10), p.5979-5990

2015

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Repeat-Associated Non-ATG (RAN) Translation in Fuchs' Endothelial Corneal Dystrophy

Investigative ophthalmology & visual science, 2018-04, Vol.59 (5), p.1888-1896

2018

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Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia

Nature chemical biology, 2006-10, Vol.2 (10), p.551-558

2006

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Epigenetic therapy for Friedreich ataxia

Annals of neurology, 2014-10, Vol.76 (4), p.489-508

2014

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HDAC inhibitor 4b ameliorates the disease phenotype and transcriptional abnormalities in Huntington's disease transgenic mice

Proceedings of the National Academy of Sciences - PNAS, 2008-10, Vol.105 (40), p.15564-15569

2008

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Histone deacetylase (HDAC) inhibitors targeting HDAC3 and HDAC1 ameliorate polyglutamine-elicited phenotypes in model systems of Huntington's disease

Neurobiology of disease, 2012-05, Vol.46 (2), p.351-361

2012

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Role of Mismatch Repair Enzymes in GAA·TTC Triplet-repeat Expansion in Friedreich Ataxia Induced Pluripotent Stem Cells

The Journal of biological chemistry, 2012-08, Vol.287 (35), p.29861-29872

2012

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HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model

PloS one, 2008-04, Vol.3 (4), p.e1958

2008

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Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

Nature chemical biology, 2010-01, Vol.6 (1), p.25-33

2010

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Prolonged treatment with pimelic o -aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model

Neurobiology of disease, 2011-06, Vol.42 (3), p.496-505

2011

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Milestones in transcription and chromatin published in the Journal of Biological Chemistry

The Journal of biological chemistry, 2019-02, Vol.294 (5), p.1652-1660

Gottesfeld, Joel M.

2019

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Quantitative Proteomic Analysis Identifies Targets and Pathways of a 2‑Aminobenzamide HDAC Inhibitor in Friedreich’s Ataxia Patient iPSC-Derived Neural Stem Cells

Journal of proteome research, 2014-11, Vol.13 (11), p.4558-4566

2014

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A non-synonymous single nucleotide polymorphism in SIRT6 predicts neurological severity in Friedreich ataxia

Frontiers in molecular biosciences, 2022-09, Vol.9, p.933788-933788

2022

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Length-dependent CTG.CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells

Human molecular genetics, 2013-12, Vol.22 (25), p.5276-5287

2013

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Mechanism of Action of 2-Aminobenzamide HDAC Inhibitors in Reversing Gene Silencing in Friedreich's Ataxia

Frontiers in neurology, 2015, Vol.6, p.44-44

2015

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Introduction to the Thematic Minireview Series: Chromatin and transcription

The Journal of biological chemistry, 2018-09, Vol.293 (36), p.13775-13777

2018

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Alkylation of duplex DNA in nucleosome core particles by duocarmycin SA and yatakemycin

Nature chemical biology, 2006-02, Vol.2 (2), p.79-82

2006

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Small molecules affecting transcription in Friedreich ataxia

Pharmacology & therapeutics (Oxford), 2007-11, Vol.116 (2), p.236-248

Gottesfeld, Joel M

2007

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Increasing frataxin gene expression with histone deacetylase inhibitors as a therapeutic approach for Friedreich's ataxia

Journal of neurochemistry, 2013-08, Vol.126 (s1), p.147-154

2013

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Molecular Recognition of the Nucleosomal "Supergroove"

Proceedings of the National Academy of Sciences - PNAS, 2004-05, Vol.101 (18), p.6864-6869

2004

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Chromatin structure determines accessibility of a hairpin polyamide–chlorambucil conjugate at histone H4 genes in pancreatic cancer cells

Bioorganic & medicinal chemistry letters, 2012-06, Vol.22 (12), p.4068-4071

2012

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Molecular Mechanisms and Therapeutics for the GAA·TTC Expansion Disease Friedreich Ataxia

The Effects of Pharmacological Inhibition of Histone Deacetylase 3 (HDAC3) in Huntington's Disease Mice

Pimelic Diphenylamide 106 Is a Slow, Tight-binding Inhibitor of Class I Histone Deacetylases

Two new pimelic diphenylamide HDAC inhibitors induce sustained frataxin upregulation in cells from Friedreich's ataxia patients and in a mouse model

RNA Toxicity and Missplicing in the Common Eye Disease Fuchs Endothelial Corneal Dystrophy

Repeat-Associated Non-ATG (RAN) Translation in Fuchs' Endothelial Corneal Dystrophy

Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia

Epigenetic therapy for Friedreich ataxia

HDAC inhibitor 4b ameliorates the disease phenotype and transcriptional abnormalities in Huntington's disease transgenic mice

Histone deacetylase (HDAC) inhibitors targeting HDAC3 and HDAC1 ameliorate polyglutamine-elicited phenotypes in model systems of Huntington's disease

Role of Mismatch Repair Enzymes in GAA·TTC Triplet-repeat Expansion in Friedreich Ataxia Induced Pluripotent Stem Cells

HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

Prolonged treatment with pimelic o -aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model

Milestones in transcription and chromatin published in the Journal of Biological Chemistry

Quantitative Proteomic Analysis Identifies Targets and Pathways of a 2‑Aminobenzamide HDAC Inhibitor in Friedreich’s Ataxia Patient iPSC-Derived Neural Stem Cells

A non-synonymous single nucleotide polymorphism in SIRT6 predicts neurological severity in Friedreich ataxia

Length-dependent CTG.CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells

Mechanism of Action of 2-Aminobenzamide HDAC Inhibitors in Reversing Gene Silencing in Friedreich's Ataxia

Introduction to the Thematic Minireview Series: Chromatin and transcription

Alkylation of duplex DNA in nucleosome core particles by duocarmycin SA and yatakemycin

Small molecules affecting transcription in Friedreich ataxia

Increasing frataxin gene expression with histone deacetylase inhibitors as a therapeutic approach for Friedreich's ataxia

Molecular Recognition of the Nucleosomal "Supergroove"

Chromatin structure determines accessibility of a hairpin polyamide–chlorambucil conjugate at histone H4 genes in pancreatic cancer cells

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