TY  - GEN
AU  - Soragni,Elisabetta
AU  - Miao,Wenyan
AU  - Iudicello,Marco
AU  - Jacoby,David
AU  - De Mercanti,Stefania
AU  - Clerico,Marinella
AU  - Longo,Filomena
AU  - Piga,Antonio
AU  - Ku,Sherman
AU  - Campau,Erica
AU  - Du,Jintang
AU  - Penalver,Pablo
AU  - Rai,Myriam
AU  - Madara,Joseph C
AU  - Nazor,Kristopher
AU  - O'Connor,Melinda
AU  - Maximov,Anton
AU  - Loring,Jeanne F
AU  - Pandolfo,Massimo
AU  - Durelli,Luca
AU  - Gottesfeld,Joel M
AU  - Rusche,James R
TI  - Epigenetic therapy for Friedreich ataxia
SN  - 1531-8249
PY  - 2014///1211
KW  - Administration, Oral
KW  - Adolescent
KW  - Adult
KW  - Aminocaproates
KW  - pharmacology
KW  - Area Under Curve
KW  - Benzamides
KW  - Cell Differentiation
KW  - drug effects
KW  - Cell Line, Transformed
KW  - Chromatin Immunoprecipitation
KW  - Cohort Studies
KW  - Cross-Sectional Studies
KW  - DNA Methylation
KW  - Dose-Response Relationship, Drug
KW  - Double-Blind Method
KW  - Female
KW  - Friedreich Ataxia
KW  - drug therapy
KW  - Gene Expression Regulation
KW  - Histone Deacetylase Inhibitors
KW  - therapeutic use
KW  - Humans
KW  - Iron-Binding Proteins
KW  - genetics
KW  - Leukocytes, Mononuclear
KW  - Male
KW  - Membrane Potentials
KW  - Middle Aged
KW  - Nerve Tissue Proteins
KW  - Neurons
KW  - Pluripotent Stem Cells
KW  - Trinucleotide Repeat Expansion
KW  - Young Adult
KW  - Frataxin
N1  - Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1002/ana.24260
ER  -