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′ 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 o...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2019-02-01
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| Series: | Brain Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2076-3425/9/2/39 |