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Promising new RNA editing technology could be used to treat genetic diseases
New research by the University of California, San Diego could provide a much simpler way to repair disease-causing mutations in RNA.
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Rett syndrome
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Investigating neural oscillations in human brain organoids
This article delves into research at the University of California Los Angeles, US, where stem-cell derived brain organoids that can mimic electrical activity have been developed.
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Brain organoids used to identify promising treatments for Rett syndrome
Two drugs, Nefiracetam and PHA 543613, were able to return neuronal signalling to near normal in organoids derived from patients with the autism spectrum disorder, Rett syndrome.
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Cancer drug could be used to treat Rett syndrome, finds study
Researchers have found that a compound named JQ1, an experimental cancer drug, extends the life of mice with Rett syndrome.
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SB216763 shows preclinical efficacy in Rett syndrome
Researchers describe how a new drug is able to reduce the symptoms and activate the dormant neurons characteristic of Rett Syndrome...
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X chromosome reactivation could treat Rett syndrome
Reactivation of the inactive X chromosome could lead to an increased expression of the healthy protein underlying the neurodevelopmental disorder...