Reduction of Fmr1 mRNA Levels Rescues Pathological Features in Cortical Neurons in a Model of FXTAS

Fragile X-associated tremor ataxia syndrome (FXTAS) is a rare disorder associated to the presence of the fragile X premutation, a 55–200 CGG repeat expansion in the 5′ UTR of the FMR1 gene. Two main neurological phenotypes have been described in carriers of the CGG premutation: (1) neurodevelopmenta...

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Pubblicato in:Mol Ther Nucleic Acids
Autori principali: Drozd, Malgorzata, Delhaye, Sébastien, Maurin, Thomas, Castagnola, Sara, Grossi, Mauro, Brau, Frédéric, Jarjat, Marielle, Willemsen, Rob, Capovilla, Maria, Hukema, Renate K., Lalli, Enzo, Bardoni, Barbara
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2019
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6838541/
https://ncbi.nlm.nih.gov/pubmed/31671347
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.09.018
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