A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis

Repeat-associated non-AUG-initiated translation of expanded CGG repeats (CGG RAN) from the FMR1 5′-leader produces toxic proteins that contribute to neurodegeneration in fragile X-associated tremor/ataxia syndrome. Here we describe how unexpanded CGG repeats and their translation play conserved role...

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Veröffentlicht in:	Nature neuroscience 2020-03, Vol.23 (3), p.386-397
Hauptverfasser:	Rodriguez, Caitlin M., Wright, Shannon E., Kearse, Michael G., Haenfler, Jill M., Flores, Brittany N., Liu, Yu, Ifrim, Marius F., Glineburg, Mary R., Krans, Amy, Jafar-Nejad, Paymaan, Sutton, Michael A., Bassell, Gary J., Parent, Jack M., Rigo, Frank, Barmada, Sami J., Todd, Peter K.
Format:	Artikel
Sprache:	eng
Schlagworte:	631/337/574 631/378/1689/2608 631/378/1689/364 631/378/2583 631/45/500 Animal Genetics and Genomics Animals Antisense oligonucleotides Ataxia Behavioral Sciences Biological Techniques Biomedical and Life Sciences Biomedicine Cell Line Cell Survival - genetics Control DNA Repeat Expansion - genetics Female FMR1 protein Fragile X Mental Retardation Protein - biosynthesis Fragile X Mental Retardation Protein - genetics Fragile X syndrome Fragile X Syndrome - genetics Genetic aspects Genetic translation Glutamic acid receptors (metabotropic) Induced Pluripotent Stem Cells Intellectual disabilities Male Mental illness Mice Neurobiology Neurodegeneration Neurons Neurons - metabolism Neurosciences Oligonucleotides Oligonucleotides, Antisense - pharmacology Physiological aspects Protein Biosynthesis Protein synthesis Proteins Rats Rats, Long-Evans Rats, Sprague-Dawley Receptor, Metabotropic Glutamate 5 - biosynthesis Receptor, Metabotropic Glutamate 5 - genetics Receptors RNA Survival Synthesis Toxicity Translation Tremor Trinucleotide Repeats - genetics
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