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https://www.selleckchem.com/products/gdc6036.html People organize and convey their thoughts according to narratives. However, neuroscientists are often reluctant to incorporate narrative stimuli into their experiments. We argue that narratives deserve wider adoption in human neuroscience because they tap into the brain's native machinery for representing the world and provide rich variability for testing hypotheses. Implanted neural probes are among the most important techniques in both fundamental and clinical neuroscience. Despite great successes and promise, neural electrodes are technically limited by their scalability. A recent study by Obaid et al. demonstrated an innovative way to greatly scale up the channel count and density of neural electrode arrays. Developmental and epileptic encephalopathy (DEE) associated with de novo variants in the gene encoding dynamin-1 (DNM1) is a severe debilitating disease with no pharmacological remedy. Like most genetic DEEs, the majority of DNM1 patients suffer from therapy-resistant seizures and comorbidities such as intellectual disability, developmental delay, and hypotonia. We tested RNAi gene therapy in the Dnm1 fitful mouse model of DEE using a Dnm1-targeted therapeutic microRNA delivered by a self-complementary adeno-associated virus vector. Untreated or control-injected fitful mice have growth delay, severe ataxia, and lethal tonic-clonic seizures by 3 weeks of age. These major impairments are mitigated following a single treatment in newborn mice, along with key underlying cellular features including gliosis, cell death, and aberrant neuronal metabolic activity typically associated with recurrent seizures. Our results underscore the potential for RNAi gene therapy to treat DNM1 disease and other genetic DEEs where treatment would require inhibition of the pathogenic gene product. Retinal pigment epithelial (RPE) cell replacement therapy has provided promising outcomes in the treatment of retinal degenerative disease
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