Clonal Rett Syndrome cell lines to test compounds for activation of wild-type MeCP2 expression

Dongbo Yu, Fuminori Sakurai, David R. Corey

Research output: Contribution to journalArticle

10 Scopus citations

Abstract

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 measuring wild-type MECP2 mRNA levels and tested small molecule epigenetic activators for their ability to activate gene expression. Although our pilot screen did not identify activators of MECP2 expression, it established the value of using clonal cells and defined challenges that must be overcome.

Original languageEnglish (US)
Pages (from-to)5202-5205
Number of pages4
JournalBioorganic and Medicinal Chemistry Letters
Volume21
Issue number18
DOIs
StatePublished - Sep 15 2011

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Keywords

  • Allele-specific cell-line
  • MeCP2
  • Nested PCR
  • Rett Syndrome
  • Reversal of X-inactivation

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Pharmaceutical Science
  • Drug Discovery
  • Clinical Biochemistry
  • Organic Chemistry

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