Epigenetic therapy for Friedreich ataxia.

Soragni, Elisabetta

Epigenetic therapy for Friedreich ataxia. [electronic resource] - Annals of neurology Oct 2014 - 489-508 p. digital

Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

1531-8249

10.1002/ana.24260 doi


Administration, Oral
Adolescent
Adult
Aminocaproates--pharmacology
Area Under Curve
Benzamides--pharmacology
Cell Differentiation--drug effects
Cell Line, Transformed
Chromatin Immunoprecipitation
Cohort Studies
Cross-Sectional Studies
DNA Methylation--drug effects
Dose-Response Relationship, Drug
Double-Blind Method
Female
Friedreich Ataxia--drug therapy
Gene Expression Regulation--drug effects
Histone Deacetylase Inhibitors--therapeutic use
Humans
Iron-Binding Proteins--genetics
Leukocytes, Mononuclear--drug effects
Male
Membrane Potentials--drug effects
Middle Aged
Nerve Tissue Proteins--genetics
Neurons--drug effects
Pluripotent Stem Cells
Trinucleotide Repeat Expansion--genetics
Young Adult
Frataxin