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

More than ~200 CGG repeats in the 5′ untranslated region of the FMR1 gene results in transcriptional silencing and the absence of the FMR1 encoded protein, FMRP. FMRP is an RNA-binding protein that regulates the transport and translation of a variety of brain mRNAs in an activity-dependent manner. T...

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Detalles Bibliográficos
Autores principales:	Kumari, Daman, Gazy, Inbal, Usdin, Karen
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2019
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6406686/ https://www.ncbi.nlm.nih.gov/pubmed/30759772 http://dx.doi.org/10.3390/brainsci9020039

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