Sage Journals: Discover world-class research

Retricted access

Research article

First published 2020-08

Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy

Jixue Li, Yanjie Li, Jun Wang [...]

Human Gene Therapy

Retricted access

Research article

First published 2018-02

Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat

Liande Li, Xiulong Shen, Zhongtian Liu [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2012-9

Clinical Monitoring in a Patient With Friedreich Ataxia and Osteogenic Sarcoma

Eric C. Deutsch, Lauren A. Seyer, Susan L. Perlman [...]

Journal of Child Neurology

Retricted access

Review article

First published 2014-08

Cell and Gene Therapy for Friedreich Ataxia: Progress to Date

Marguerite V. Evans-Galea, Alice Pébay, Mirella Dottori [...]

Human Gene Therapy

Retricted access

Research article

First published 2012-9

Cardiomyopathy in Friedreich Ataxia

R. Mark Payne, Gregory R. Wagner

Journal of Child Neurology

Retricted access

Research article

First published 2023-07

Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

Carlos Munoz-Zuluaga, Monica Gertz, Melissa Yost-Bido [...]

Human Gene Therapy

Retricted access

Research article

First published 2012-9

Novel Diagnostic Paradigms for Friedreich Ataxia

Karlla W. Brigatti, Eric C. Deutsch, David R. Lynch [...]

Journal of Child Neurology

Retricted access

Research article

First published 2012-9

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

Elisabetta Soragni, Chunping Xu, Heather L. Plasterer [...]

Journal of Child Neurology

Retricted access

Research article

First published 2016-12

Alleviating GAA Repeat Induced Transcriptional Silencing of the Friedreich's Ataxia Gene During Somatic Cell Reprogramming

Urszula Polak, Yanjie Li, Jill Sergesketter Butler [...]

Stem Cells and Development

Retricted access

Research article

First published 2016-7

Clinical Experience With Deferiprone Treatment for Friedreich Ataxia

Sandra Elincx-Benizri, Amir Glik, Drorit Merkel [...]

Journal of Child Neurology

Search results