Rationale for the Development of 2-Aminobenzamide Histone Deacetylase Inhibitors as Therapeutics for Friedreich Ataxia

Preventing the ubiquitin–proteasome-dependent degradation of frataxin, the protein defective in Friedreich's ataxia

(2011) Alessandra Rufini et al.   HUMAN MOLECULAR GENETICS

A TAT–Frataxin fusion protein increases lifespan and cardiac function in a conditional Friedreich's ataxia mouse model

(2011) Piyush M. Vyas et al.   HUMAN MOLECULAR GENETICS

Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study

(2011) Thomas Meier et al.   JOURNAL OF NEUROLOGY

Mortality in Friedreich Ataxia

(2011) Amy Y. Tsou et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

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

(2011) Chiranjeevi Sandi et al.   NEUROBIOLOGY OF DISEASE

Hyperexpansion of GAA repeats affects post-initiation steps of FXN transcription in Friedreich's ataxia

(2011) E. Kim et al.   NUCLEIC ACIDS RESEARCH

Human Iron−Sulfur Cluster Assembly, Cellular Iron Homeostasis, and Disease

(2010) Hong Ye et al.   BIOCHEMISTRY

Combined Therapy with Idebenone and Deferiprone in Patients with Friedreich’s Ataxia

(2010) Daniel Velasco-Sánchez et al.   CEREBELLUM

Long intronic GAA repeats causing Friedreich ataxia impede transcription elongation

(2010) Tanel Punga et al.   EMBO Molecular Medicine

Understanding the molecular mechanisms of Friedreich's ataxia to develop therapeutic approaches

(2010) S. Schmucker et al.   HUMAN MOLECULAR GENETICS

Repeat Expansion Affects Both Transcription Initiation and Elongation in Friedreich Ataxia Cells

(2010) Daman Kumari et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Two New Pimelic Diphenylamide HDAC Inhibitors Induce Sustained Frataxin Upregulation in Cells from Friedreich's Ataxia Patients and in a Mouse Model

(2010) Myriam Rai et al.   PLoS One

Chemical Probes Identify a Role for Histone Deacetylase 3 in Friedreich's Ataxia Gene Silencing

(2009) Chunping Xu et al.   CHEMISTRY & BIOLOGY

Epigenetic Silencing in Friedreich Ataxia Is Associated with Depletion of CTCF (CCCTC-Binding Factor) and Antisense Transcription

(2009) Irene De Biase et al.   PLoS One

Influence of Friedreich Ataxia GAA Noncoding Repeat Expansions on Pre-mRNA Processing

(2008) Marco Baralle et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Recombinant Human Erythropoietin Increases Frataxin Protein Expression Without Increasing mRNA Expression

(2008) Fabio Acquaviva et al.   CEREBELLUM

Chromatin Remodeling in the Noncoding Repeat Expansion Diseases

(2008) Daman Kumari et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2008) C. James Chou et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Therapeutic application of histone deacetylase inhibitors for central nervous system disorders

(2008) Aleksey G. Kazantsev et al.   NATURE REVIEWS DRUG DISCOVERY

HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model

(2008) Myriam Rai et al.   PLoS One

The Antisense Transcriptomes of Human Cells

(2008) Yiping He et al.   SCIENCE

Exploration of the internal cavity of histone deacetylase (HDAC) with selective HDAC1/HDAC2 inhibitors (SHI-1:2)

(2007) Joey L. Methot et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS