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Rett Syndrome: Crossing the Threshold to Clinical Translation

author

Debashish Menon

Adrian Peter Bird

1947 -

published in: Trends in Neurosciences

date of publication: 2016-02

language: English

main subject: Rett syndrome, clinic

Cites articles 124

Date

Title

Authors

2016

To edit or not to edit: regulation of ADAR editing specificity and efficiency
2016-02

Advances in therapeutic CRISPR/Cas9 genome editing

Nataša Savić, Gerald Schwank
2015-08-03

MECP2 disorders: from the clinic to mice and back

Laura Marie Lombardi, Huda Zoghbi
2015-07-01

Rett Syndrome: Reaching for Clinical Trials

Lucas Pozzo-miller, Alan K. Percy
2015-05-14

Drug Discovery of Therapies for Duchenne Muscular Dystrophy
2015-04-13

MeCP2 binds to non-CG methylated DNA as neurons mature, influencing transcription and the timing of onset for Rett syndrome

Huda Zoghbi
2015-03-30

Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa

Rachel Jones, Claudia Bracaglia, Pavla Doležalová, Rolando Cimaz
2015-03-11

Disruption of DNA-methylation-dependent long gene repression in Rett syndrome

Martin Hemberg
2015-03-03

Rett syndrome: a complex disorder with simple roots

Matthew J Lyst, Adrian Peter Bird
2015-02-07

Mechanisms of Functional Hypoconnectivity in the Medial Prefrontal Cortex of Mecp2 Null Mice.

Addison C Enomoto
2015-08-24

Chronic Administration of the N-Methyl-D-Aspartate Receptor Antagonist Ketamine Improves Rett Syndrome Phenotype.
2014-03-10

MeCP2 suppresses nuclear microRNA processing and dendritic growth by regulating the DGCR8/Drosha complex

Wenqing Xu
2014-12-24

Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative

Isabelle Ray-Coquard, Jean-Yves Blay, Martin McCabe, Emma Hall, Lucinda J. Billingham, Matthew R Sydes, Dirk Arnold, Helena Earl, Lisa Licitra, Serge Leyvraz, Jeremy S Whelan, Corneel Coens
2014-10-31

BDNF release is required for the behavioral actions of ketamine
2014-12-01

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.

Amanda J Ward
2014-09-25

Excitation/inhibition imbalance and impaired synaptic inhibition in hippocampal area CA3 of Mecp2 knockout mice

Lucas Pozzo-miller
2014-08-18

Genetic and pharmacological reactivation of the mammalian inactive X chromosome

Sanchita Bhatnagar, Jianhong Ou
2014-07-22

Developmental delay in Rett syndrome: data from the natural history study

Jeffrey L. Neul, Alan K. Percy, Walter E Kaufmann, Daniel G Glaze
2014-04-01

Methodology of clinical trials for rare diseases

Paula Williamson, Catrin Tudur Smith
2014-06-26

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

Huda Zoghbi, Maria H Chahrour
2014-04-18

Therapeutics based on stop codon readthrough
2014-04-21

MeCP2: the long trip from a chromatin protein to neurological disorders

Manel Esteller
2014-03-12

Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome

Ingrid A Holm, Walter E Kaufmann
2014-01-12

Effects of ω-3 PUFAs supplementation on myocardial function and oxidative stress markers in typical Rett syndrome

Silvia Maffei, Pierpaolo Cannarile, Silvia Leoncini, Cinzia Signorini, Alessandra Pecorelli, Lucia Ciccoli, Thierry Durand, Joussef Hayek
2014-01-07

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

Vishnu Anand Cuddapah, Daniel Tarquinio, Michelle L. Olsen, Alan K. Percy, Walter E Kaufmann, Jeffrey L. Neul, Daniel G Glaze
2014-10-09

Conditional deletion of Mecp2 in parvalbumin-expressing GABAergic cells results in the absence of critical period plasticity.
2014-09-24

Communication intervention in Rett syndrome: a survey of speech language pathologists in Swedish health services
2013-07-01

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

Flavia de Lima Alves, Juri Rappsilber, Robert Ekiert, Jim Selfridge, Adrian Peter Bird, Jacky Guy
2013-12-22

Distribution, recognition and regulation of non-CpG methylation in the adult mammalian brain

Guoping Fan, Guo-Li Ming, Hongjun Song, Hongda Li, Jaehoon Shin, Joo Heon Shin
2013-01-01

Recent Progress in Rett Syndrome and MeCP2 Dysfunction: Assessment of Potential Treatment Options

Lucas Pozzo-miller, Alan K. Percy
2013-11-05

Strategies for postmarketing surveillance of drugs for rare diseases

Aaron S Kesselheim, Joshua J Gagne
2013-10-01

Global transcriptional and translational repression in human-embryonic-stem-cell-derived Rett syndrome neurons

Rudolf Jaenisch, Yun Li, Julien Muffat, Dirk Hockemeyer, Richard A. Young, Maisam Mitalipova, Jakob Lovén, Haoyi Wang

Article - wd:Q26772909

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