Article
Clonal Rett Syndrome cell lines to test compounds for activation of wild-type MeCP2 expression.
Bioorganic & medicinal chemistry letters - 15 Sept 2011
Yu Dongbo, Sakurai Fuminori, Corey David R
Abstract excerpt
Rett Syndrome is an X-linked progressive neurological disorder caused by inactivation of one allele of the MECP2 gene. There are no curative treatments, and activation of wild-type MECP2 expression is one strategy for stabilizing or reversing the disease. We isolated fibroblast clones that express exclusively either the wild-type or a 32-bp-deletion mutant form of MECP2. We developed a sensitive assay for...
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