Cell Rep: A new breakthrough, RNA editing can correct protein mutations and reverse neurological diseases.
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Last Update: 2020-07-27
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Source: Internet
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Author: User
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!---- Introduction: Gene editing is a new and more accurate genetic engineering technique that modifies specific target genes in an organism's genomegene editing technology is the ability to allow humans to "edit" target genes to modify specific nucleic acid fragments, researchers at Oregon Health and Science University have made a breakthrough in gene editingscientists succeeded in editing RNA in living animals and then repaired RNA corrected a mutation in a protein that causes a neurological disease called Rett syndromeresearchers at Oregon Health and Science University published their progress in the July 14 issue of cell reports"This is the first example of using programmable RNA editing to repair genes in mouse models of neurological diseases, and provides us with an attractive approach," said senior scientist and senior author DrGail Mandel of the Vollum Institute at Oregon Health and Science University," Reiter syndrome is a neurodebilitating disorder caused by a mutation in the gene that encodes the MeCP2 proteinthe disease occurs almost exclusively in girls, and because it is located on the X chromosome, it is estimated to affect one in every 10,000 live birthsThe abstract, led by DrJohn Sinnamon, Mandel and his co-authors have been working to repair the mutated MeCP2 protein, the RNA, which acts as a messenger, and carries DNA commands to control protein synthesisother scientists have used programmable RNA editing repair techniques to treat muscular dystrophy and even hearing loss in genetically engineered mice, however, this is the first evidence that the technique is promising in neurological disorders, rooted in genetic mutations in thousands of different cell types in the brainnervous system challenges the technique more than other organs, such as muscles or livers, because they have much less cellular heterogeneitythis new study targets and repairs a variety of cell types of MeCP2 proteins, the first in science"We fixed the MeCP2 protein in three different groups of neurons, so if we could spread the editing ingredients widely, it might work throughout the brain," saysMandel " the results of the study showed 50% editing consistency, and meCP2 proteins and heterogeneous chromatins had comparable correlations in several hippocampus neuronal subtypes the results suggest that the entire brain's neuronal population should have a similar rate of repair through peripheral injections is possible through peripheral injections, comprehensive behavioral testing combined with quantitative measurements of MeCP2 protein function and gene expression, and needs to be done in mouse models of male and female Rett syndrome in mice, it is not clear how much MeCP2 each cell needs to repair, and how many neurons and glial cells need to be repaired to reverse the Rett syndrome phenotype the effective editing of Mecp2 RNA after the male hippocampus was injected with Mecp2, a landmark study led by Dr Adrian Bird of the University of Edinburgh suggests that it is possible to reverse recourse to reed-like symptoms in mice, suggesting that it is also possible in humans Although research by Oregon Health and Science University shows that RNA repair promises to be evidence of the concept.
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