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Pharmacological Reactivation of the Silenced <i>FMR1</i> Gene as a Targeted Therapeutic Approach for Fragile X Syndrome

More than ~200 CGG repeats in the 5&#8242; untranslated region of the <i>FMR1</i> gene results in transcriptional silencing and the absence of the <i>FMR1</i> encoded protein, FMRP. FMRP is an RNA-binding protein that regulates the transport and translation of a variety of brain mRNAs in an activity...

Ausführliche Beschreibung

Gespeichert in:
Hauptverfasser: Daman Kumari, Inbal Gazy, Karen Usdin
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI AG 2019-02-01
Schriftenreihe:Brain Sciences
Schlagworte:
fragile X syndrome
gene reactivation
RNA:DNA hybrid
FMRP
histone methylation
DNA methylation
<i>FMR1</i>
PRC2
Online-Zugang:https://www.mdpi.com/2076-3425/9/2/39
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