Long intronic GAA repeats causing Friedreich ataxia impede transcription elongation

EMBO Molecular Medicine DOI 10.1002/emmm.201000064
Volume 2 Issue 4,;Pages 120-129, Published Online: 1 Apr 2010

Tanel Punga, Marc Bühler

Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland

Keywords: epigenetics • Friedreich ataxia (FRDA) • heterochromatic gene silencing • histone modification • triplet repeat expansion disorder (TRED)

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Cell and gene therapies: moving from research to clinic

Journal of Translational Medicine 2010, 8:31 (29 March 2010)

Stroncek DF, Puri RK
1Department of Transfusion Medicine, Clinical Center, NIH, Bethesda, Maryland, USA
2Center for Biologics Evaluation and Research, Food and Drug Administration,
Bethesda, Maryland, USA

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