Site-directed RNA repair of endogenous Mecp2 RNA in neurons

MeCP2 recognizes cytosine methylated tri-nucleotide and di-nucleotide sequences to tune transcription in the mammalian brain

(2017) Sabine Lagger et al.   PLoS Genetics

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

(2017) Sarah E. Sinnett et al.   Molecular Therapy-Methods & Clinical Development

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

(2017) Kamal K.E. Gadalla et al.   Molecular Therapy-Methods & Clinical Development

Binding Analysis of Methyl-CpG Binding Domain of MeCP2 and Rett Syndrome Mutations

(2016) Ye Yang et al.   ACS Chemical Biology

Adeno-associated Virus (AAV) Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of AAV Serotypes 4, 5, and 11

(2016) Lauriel F. Earley et al.   JOURNAL OF VIROLOGY

Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity

(2016) Melissa M Matthews et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

An efficient system for selectively altering genetic information within mRNAs

(2016) Maria Fernanda Montiel-González et al.   NUCLEIC ACIDS RESEARCH

Probing RNA recognition by human ADAR2 using a high-throughput mutagenesis method

(2016) Yuru Wang et al.   NUCLEIC ACIDS RESEARCH

Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity

(2016) Melissa M Matthews et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

A High-Resolution Imaging Approach to Investigate Chromatin Architecture in Complex Tissues

(2015) Michael W. Linhoff et al.   CELL

The molecular basis of variable phenotypic severity among common missense mutations causing Rett syndrome

(2015) Kyla Brown et al.   HUMAN MOLECULAR GENETICS

Site-Directed RNA Editing in Vivo Can Be Triggered by the Light-Driven Assembly of an Artificial Riboprotein

(2015) Alfred Hanswillemenke et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

A-to-I editing of coding and non-coding RNAs by ADARs

(2015) Kazuko Nishikura   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Recognition of duplex RNA by the deaminase domain of the RNA editing enzyme ADAR2

(2015) Kelly J. Phelps et al.   NUCLEIC ACIDS RESEARCH

Improving Site-Directed RNA Editing In Vitro and in Cell Culture by Chemical Modification of the GuideRNA

(2014) Paul Vogel et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Site-Directed RNA Editing with Antagomir Deaminases - A Tool to Study Protein and RNA Function

(2014) Paul Vogel et al.   ChemMedChem

Methyl-CpG-binding protein 2(MECP2) mutation type is associated with disease severity in Rett syndrome

(2014) Vishnu Anand Cuddapah et al.   JOURNAL OF MEDICAL GENETICS

Cell-Type-Specific Repression by Methyl-CpG-Binding Protein 2 Is Biased toward Long Genes

(2014) K. Sugino et al.   JOURNAL OF NEUROSCIENCE

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Optimal guideRNAs for re-directing deaminase activity of hADAR1 and hADAR2 in trans

(2014) Marius F. Schneider et al.   NUCLEIC ACIDS RESEARCH

Rett-causing mutations reveal two domains critical for MeCP2 function and for toxicity in MECP2 duplication syndrome mice

(2014) Laura Dean Heckman et al.   eLife

Global Transcriptional and Translational Repression in Human-Embryonic-Stem-Cell-Derived Rett Syndrome Neurons

(2013) Yun Li et al.   Cell Stem Cell

Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome

(2013) S. K. Garg et al.   JOURNAL OF NEUROSCIENCE

Rett syndrome mutations abolish the interaction of MeCP2 with the NCoR/SMRT co-repressor

(2013) Matthew J Lyst et al.   NATURE NEUROSCIENCE

Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing

(2013) M. F. Montiel-Gonzalez et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Morphological and functional reversal of phenotypes in a mouse model of Rett syndrome

(2012) Lianne Robinson et al.   BRAIN

Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice

(2012) Kamal KE Gadalla et al.   MOLECULAR THERAPY

MeCP2 Mutation Results in Compartment-Specific Reductions in Dendritic Branching and Spine Density in Layer 5 Motor Cortical Neurons of YFP-H Mice

(2012) David P. Stuss et al.   PLoS One

Mechanistic insights into editing-site specificity of ADARs

(2012) A. Kuttan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A role for glia in the progression of Rett’s syndrome

(2011) Daniel T. Lioy et al.   NATURE

Rett syndrome mutation MeCP2 T158A disrupts DNA binding, protein stability and ERP responses

(2011) Darren Goffin et al.   NATURE NEUROSCIENCE

Predicting sites of ADAR editing in double-stranded RNA

(2011) Julie M. Eggington et al.   Nature Communications

Rett syndrome: Revised diagnostic criteria and nomenclature

(2010) Jeffrey L. Neul et al.   ANNALS OF NEUROLOGY

Rett syndrome diagnostic criteria: Lessons from the Natural History Study

(2010) Alan K. Percy et al.   ANNALS OF NEUROLOGY

Functions and Regulation of RNA Editing by ADAR Deaminases

(2010) Kazuko Nishikura   Annual Review of Biochemistry

Neuronal MeCP2 Is Expressed at Near Histone-Octamer Levels and Globally Alters the Chromatin State

(2010) Peter J. Skene et al.   MOLECULAR CELL

Severe congenital encephalopathy caused by MECP2 null mutations in males: central hypoxia and reduced neuronal dendritic structure

(2009) B Schüle et al.   CLINICAL GENETICS

Widespread changes in dendritic and axonal morphology inMecp2-mutant mouse models of rett syndrome: Evidence for disruption of neuronal networks

(2009) Pavel V. Belichenko et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Non–cell autonomous influence of MeCP2-deficient glia on neuronal dendritic morphology

(2009) Nurit Ballas et al.   NATURE NEUROSCIENCE

Evidence for both neuronal cell autonomous and nonautonomous effects of methyl-CpG-binding protein 2 in the cerebral cortex of female mice with Mecp2 mutation

(2009) Nadia P. Belichenko et al.   NEUROBIOLOGY OF DISEASE

Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice

(2009) Daniela Tropea et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA