MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

Mouse models of MeCP2 disorders share gene expression changes in the cerebellum and hypothalamus

(2009) Shay Ben-Shachar et al.   HUMAN MOLECULAR GENETICS

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency

(2009) Akitsu Hotta et al.   NATURE METHODS

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

(2009) Nurit Ballas et al.   NATURE NEUROSCIENCE

A methyl-CpG-binding protein 2–enhanced green fluorescent protein reporter mouse model provides a new tool for studying the neuronal basis of Rett syndrome

(2009) Ralf S. Schmid et al.   NEUROREPORT

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL

Rett Syndrome-causing Mutations in Human MeCP2 Result in Diverse Structural Changes That Impact Folding and DNA Interactions

(2008) Rajarshi P. Ghosh et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Deletion of Mecp2 in Sim1-Expressing Neurons Reveals a Critical Role for MeCP2 in Feeding Behavior, Aggression, and the Response to Stress

(2008) Sharyl L. Fyffe et al.   NEURON

MeCP2, a Key Contributor to Neurological Disease, Activates and Represses Transcription

(2008) M. Chahrour et al.   SCIENCE

Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons

(2008) J. T. Dimos et al.   SCIENCE

β-Globin LCR and Intron Elements Cooperate and Direct Spatial Reorganization for Gene Therapy

(2008) Alla Buzina et al.   PLoS Genetics