Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Effect of acute and long-term administration of gold nanoparticles on biochemical parameters in rat brain

(2017) Gabriela K. Ferreira et al.   Materials Science & Engineering C-Materials for Biological Applications

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

(2017) Brett T Staahl et al.   NATURE BIOTECHNOLOGY

Public Health Literature Review of Fragile X Syndrome

(2017) Melissa Raspa et al.   PEDIATRICS

Behavioral Phenotyping Assays for Genetic Mouse Models of Neurodevelopmental, Neurodegenerative, and Psychiatric Disorders

(2017) Stacey J. Sukoff Rizzo et al.   Annual Review of Animal Biosciences

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Pharmacotherapy for mental health problems in people with intellectual disability

(2016) Na Young Ji et al.   CURRENT OPINION IN PSYCHIATRY

Negative Allosteric Modulation of mGluR5 Partially Corrects Pathophysiology in a Mouse Model of Rett Syndrome

(2016) Jifang Tao et al.   JOURNAL OF NEUROSCIENCE

Gene-editing therapy for neurological disease

(2016) Moira A. McMahon et al.   Nature Reviews Neurology

Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome

(2016) Nina Xie et al.   PLoS One

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases

(2015) Kentaro Ishida et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex

(2015) Akiya Watakabe et al.   NEUROSCIENCE RESEARCH

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons

(2015) Chul-Yong Park et al.   Cell Reports

Behavioral analysis of male and female Fmr1 knockout mice on C57BL/6 background

(2014) Qi Ding et al.   BEHAVIOURAL BRAIN RESEARCH

Genome editing assessment using CRISPR Genome Analyzer (CRISPR-GA)

(2014) M. Guell et al.   BIOINFORMATICS

Repetitive behaviors in the Shank1 knockout mouse model for autism spectrum disorder: Developmental aspects and effects of social context

(2014) A. Özge Sungur et al.   JOURNAL OF NEUROSCIENCE METHODS

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

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Improved vectors and genome-wide libraries for CRISPR screening

(2014) Neville E Sanjana et al.   NATURE METHODS

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Psychopharmacological Interventions in Autism Spectrum Disorder

(2014) Laura C. Politte et al.   HARVARD REVIEW OF PSYCHIATRY

Autism genetics

(2013) Antonio M. Persico et al.   BEHAVIOURAL BRAIN RESEARCH

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Instantaneous and Quantitative Functionalization of Gold Nanoparticles with Thiolated DNA Using a pH-Assisted and Surfactant-Free Route

(2012) Xu Zhang et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Negative Allosteric Modulation of the mGluR5 Receptor Reduces Repetitive Behaviors and Rescues Social Deficits in Mouse Models of Autism

(2012) J. L. Silverman et al.   Science Translational Medicine

Glia: an emerging target for neurological disease therapy

(2012) Akshata A Almad et al.   Stem Cell Research & Therapy

Modifying behavioral phenotypes in Fmr1KO mice: genetic background differences reveal autistic-like responses

(2011) Corinne M. Spencer et al.   Autism Research

Driving Opposing Behaviors with Ensembles of Piriform Neurons

(2011) Gloria B. Choi et al.   CELL

Internally Generated Preactivation of Single Neurons in Human Medial Frontal Cortex Predicts Volition

(2011) Itzhak Fried et al.   NEURON

Bidirectional Regulation of Dendritic Voltage-Gated Potassium Channels by the Fragile X Mental Retardation Protein

(2011) Hye Young Lee et al.   NEURON

Epigenetic Modification of the FMR1 Gene in Fragile X Syndrome Is Associated with Differential Response to the mGluR5 Antagonist AFQ056

(2011) S. Jacquemont et al.   Science Translational Medicine

Introduction of stearoyl moieties into a biocompatible cationic polyaspartamide derivative, PAsp(DET), with endosomal escaping function for enhanced siRNA-mediated gene knockdown

(2010) Hyun Jin Kim et al.   JOURNAL OF CONTROLLED RELEASE

Hypersensitivity to mGluR5 and ERK1/2 Leads to Excessive Protein Synthesis in the Hippocampus of a Mouse Model of Fragile X Syndrome

(2010) E. K. Osterweil et al.   JOURNAL OF NEUROSCIENCE

Size-dependent impairment of cognition in mice caused by the injection of gold nanoparticles

(2010) Yu-Shiun Chen et al.   NANOTECHNOLOGY

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE

Marble burying reflects a repetitive and perseverative behavior more than novelty-induced anxiety

(2009) Alexia Thomas et al.   PSYCHOPHARMACOLOGY

Role for metabotropic glutamate receptor 5 (mGluR5) in the pathogenesis of fragile X syndrome

(2008) Gül Dölen et al.   JOURNAL OF PHYSIOLOGY-LONDON

Polyplexes from Poly(aspartamide) Bearing 1,2-Diaminoethane Side Chains Induce pH-Selective, Endosomal Membrane Destabilization with Amplified Transfection and Negligible Cytotoxicity

(2008) Kanjiro Miyata et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Autism-like behavioral phenotypes in BTBR T+tf/J mice

(2007) H. G. McFarlane et al.   GENES BRAIN AND BEHAVIOR