DEVELOPMENTAL NEUROSCIENCEA methyl-CpG-binding protein 2–enhanced green fluorescent protein reporter mouse model provides a new tool for studying the neuronal basis of Rett syndromeSchmid, Ralf S.c; Tsujimoto, Naomid; Qu, Qianga; Lei, Honga; Li, Enb; Chen, Taipingb; Blaustein, Cecile SpielewoyaAuthor Information aDevelopmental and Molecular Pathways bEpigenetics, Novartis Institutes for Biomedical Research, Cambridge, Massachusetts cDuke University, Center for Drug Discovery and Department of Neurobiology, Durham, North Carolina dThe Harvard Stem Cell Institute, Harvard University, Cambridge, Massachusetts, USA Correspondence to Dr Cecile Spielewoy Blaustein, PhD, Developmental & Molecular Pathways, Novartis Institutes for Biomedical Research, 100 Technology Square, Office 8401, Cambridge, MA 02139, USA Tel: +1 617 871 3646; fax: +1 617 871 7066; e-mail: [email protected] Ralf S. Schmid and Naomi Tsujimoto contributed equally to this work. Received 2 November 2007; accepted 11 December 2007 NeuroReport: March 5, 2008 - Volume 19 - Issue 4 - p 393-398 doi: 10.1097/WNR.0b013e3282f5661c Buy Metrics Abstract Rett syndrome, a pervasive X-linked neurodevelopmental disorder in young girls, is caused by loss-of-function mutations in the gene that encodes the transcriptional repressor methyl-CpG-binding protein 2 (MeCP2). Mecp2-knockout mice phenocopy the major symptoms found in human patients and have advanced our understanding of the function of MeCP2 and mechanism of Rett syndrome. To study the behavior of the MeCP2 protein in vivo, we have generated a knock-in reporter mouse model that expresses MeCP2–enhanced green fluorescent protein (EGFP) fusion protein instead of endogenous MeCP2. Here we show that expression of the fusion protein in the brain remarkably mirrors endogenous MeCP2 expression in all temporal and spatial aspects. This mouse model may be a valuable tool for studying Rett syndrome and for developing therapies. © 2008 Lippincott Williams & Wilkins, Inc.