Published in Mol Ther Methods Clin Dev on April 19, 2017
Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet (1999) 22.84
Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in mice. Nat Genet (2001) 8.64
Reversal of neurological defects in a mouse model of Rett syndrome. Science (2007) 7.09
Mild overexpression of MeCP2 causes a progressive neurological disorder in mice. Hum Mol Genet (2004) 4.45
Homeostatic regulation of MeCP2 expression by a CREB-induced microRNA. Nat Neurosci (2007) 3.81
Insight into Rett syndrome: MeCP2 levels display tissue- and cell-specific differences and correlate with neuronal maturation. Hum Mol Genet (2002) 3.12
Comprehensive expression analyses of neural cell-type-specific miRNAs identify new determinants of the specification and maintenance of neuronal phenotypes. J Neurosci (2013) 2.08
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum Gene Ther (2011) 1.90
The major form of MeCP2 has a novel N-terminus generated by alternative splicing. Nucleic Acids Res (2004) 1.79
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther (2013) 1.73
Rett syndrome mutation MeCP2 T158A disrupts DNA binding, protein stability and ERP responses. Nat Neurosci (2011) 1.53
Cerebellar gene expression profiles of mouse models for Rett syndrome reveal novel MeCP2 targets. BMC Med Genet (2007) 1.49
Rett syndrome: a complex disorder with simple roots. Nat Rev Genet (2015) 1.40
Ischemic preconditioning potentiates the protective effect of stem cells through secretion of exosomes by targeting Mecp2 via miR-22. PLoS One (2014) 1.32
Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. J Neurosci (2013) 1.26
The impact of MeCP2 loss- or gain-of-function on synaptic plasticity. Neuropsychopharmacology (2012) 1.22
Identification of cis-regulatory elements for MECP2 expression. Hum Mol Genet (2006) 1.18
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Mol Ther (2012) 1.16
Morphological and functional reversal of phenotypes in a mouse model of Rett syndrome. Brain (2012) 1.14
Longevity in Rett syndrome: analysis of the North American Database. J Pediatr (2010) 1.10
Manufacturing of recombinant adeno-associated viral vectors for clinical trials. Mol Ther Methods Clin Dev (2016) 1.05
Decitabine alters the expression of Mecp2 isoforms via dynamic DNA methylation at the Mecp2 regulatory elements in neural stem cells. Mol Autism (2013) 0.98
Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration. Curr Protoc Neurosci (2011) 0.97
Loss of MeCP2 function is associated with distinct gene expression changes in the striatum. Neurobiol Dis (2013) 0.93
Alternative polyadenylation of MeCP2: Influence of cis-acting elements and trans-acting factors. RNA Biol (2010) 0.91
Mild overexpression of Mecp2 in mice causes a higher susceptibility toward seizures. Am J Pathol (2013) 0.91
Brain-derived neurotrophic factor and Rett syndrome. Handb Exp Pharmacol (2014) 0.87
Rett syndrome and the urge of novel approaches to study MeCP2 functions and mechanisms of action. Neurosci Biobehav Rev (2014) 0.86
MicroRNA-22 regulates smooth muscle cell differentiation from stem cells by targeting methyl CpG-binding protein 2. Arterioscler Thromb Vasc Biol (2015) 0.84
Visualization of spinal afferent innervation in the mouse colon by AAV8-mediated GFP expression. Neurogastroenterol Motil (2012) 0.84
Recent Progress in Rett Syndrome and MeCP2 Dysfunction: Assessment of Potential Treatment Options. Future Neurol (2013) 0.84
Developmental Dynamics of Rett Syndrome. Neural Plast (2016) 0.82
MicroRNAs downregulated in neuropathic pain regulate MeCP2 and BDNF related to pain sensitivity. FEBS Open Bio (2015) 0.80
A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome. Neurobiol Dis (2016) 0.78
Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome. Mol Ther Methods Clin Dev (2017) 0.77
Reciprocal regulation of autism-related genes MeCP2 and PTEN via microRNAs. Sci Rep (2016) 0.77
Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome. Mol Ther Methods Clin Dev (2017) 0.77