Severe offtarget effects following intravenous delivery of AAV9-MECP2 in a female mouse model of Rett syndrome

Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome

(2020) Mirko Luoni et al.   eLife

Defective proteasome biogenesis into skin fibroblasts isolated from Rett syndrome subjects with MeCP2 non-sense mutations

(2020) Diego Sbardella et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Letter to the Editor

(2020) Perry B. Shieh et al.   HUMAN GENE THERAPY

Disturbed redox homeostasis and oxidative stress: Potential players in the developmental regression in Rett syndrome

(2019) Michael Müller   NEUROSCIENCE AND BIOBEHAVIORAL REVIEWS

The course of awake breathing disturbances across the lifespan in Rett syndrome

(2018) Daniel C. Tarquinio et al.   BRAIN & DEVELOPMENT

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

OUP accepted manuscript

(2018)   HUMAN MOLECULAR GENETICS

Radically truncated MeCP2 rescues Rett syndrome-like neurological defects

(2017) Rebekah Tillotson et al.   NATURE

A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome

(2017) Valerie Matagne et al.   NEUROBIOLOGY OF DISEASE

Retention of Mitochondria in Mature Human Red Blood Cells as the Result of Autophagy Impairment in Rett Syndrome

(2017) Diego Sbardella et al.   Scientific Reports

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

Regulation of mRNA splicing by MeCP2 via epigenetic modifications in the brain

(2017) Tian-Lin Cheng et al.   Scientific Reports

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments

(2016) Amir Mehdi Ansari et al.   Stem Cell Reviews and Reports

Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments

(2016) Amir Mehdi Ansari et al.   Stem Cell Reviews and Reports

Targeting transgene to the heart and liver with AAV9 by different promoters

(2015) Bang-Dang Chen et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Disruption of MeCP2 attenuates circadian rhythm in CRISPR/Cas9-based Rett syndrome model mouse

(2015) Yoshiki Tsuchiya et al.   GENES TO CELLS

MECP2 disorders: from the clinic to mice and back

(2015) Laura Marie Lombardi et al.   JOURNAL OF CLINICAL INVESTIGATION

Rett syndrome: a complex disorder with simple roots

(2015) Matthew J. Lyst et al.   NATURE REVIEWS GENETICS

Circadian rhythm disruption in a mouse model of Rett syndrome circadian disruption in RTT

(2015) Quan Li et al.   NEUROBIOLOGY OF DISEASE

Elimination of contaminating cap genes in AAV vector virions reduces immune responses and improves transgene expression in a canine gene therapy model

(2014) Z Wang et al.   GENE THERAPY

Cellular unfolded protein response against viruses used in gene therapy

(2014) Dwaipayan Sen et al.   Frontiers in Microbiology

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

Activation of the Cellular Unfolded Protein Response by Recombinant Adeno-Associated Virus Vectors

(2013) Balaji Balakrishnan et al.   PLoS One

Preclinical research in Rett syndrome: setting the foundation for translational success

(2012) D. M. Katz et al.   Disease Models & Mechanisms

Female Mecp2+/− mice display robust behavioral deficits on two different genetic backgrounds providing a framework for pre-clinical studies

(2012) Rodney C. Samaco et al.   HUMAN MOLECULAR GENETICS

A Mouse Model for MeCP2 Duplication Syndrome: MeCP2 Overexpression Impairs Learning and Memory and Synaptic Transmission

(2012) E. S. Na et al.   JOURNAL OF NEUROSCIENCE

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

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

(2011) J H McIntosh et al.   GENE THERAPY

Expression of GFP can influence aging and climbing ability in Drosophila

(2011) R.M.S. Mawhinney et al.   GENETICS AND MOLECULAR RESEARCH

Complexities of Rett Syndrome and MeCP2

(2011) R. C. Samaco et al.   JOURNAL OF NEUROSCIENCE

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Rett syndrome: Revised diagnostic criteria and nomenclature

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

Progressive motor and respiratory metabolism deficits in post-weaning Mecp2-null male mice

(2010) Michel Pratte et al.   BEHAVIOURAL BRAIN RESEARCH

Production, Purification and Characterization of Adeno-Associated Vectors

(2010) Eduard Ayuso et al.   CURRENT GENE THERAPY

Elevated expression of MeCP2 in cardiac and skeletal tissues is detrimental for normal development

(2010) Matías Alvarez-Saavedra et al.   HUMAN MOLECULAR GENETICS

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Rett syndrome: clinical peculiarities and biological mysteries

(2008) B Hagberg   ACTA PAEDIATRICA

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY