Modeling the complex genetic architectures of brain disease

Assembling human brain organoids

(2019) Sergiu P. Paşca   SCIENCE

Induced pluripotent stem cells in disease modelling and drug discovery

(2019) R. Grant Rowe et al.   NATURE REVIEWS GENETICS

Differentiation and maturation of oligodendrocytes in human three-dimensional neural cultures

(2019) Rebecca M. Marton et al.   NATURE NEUROSCIENCE

Gene expression imputation across multiple brain regions provides insights into schizophrenia risk

(2019) Laura M. Huckins et al.   NATURE GENETICS

Engineered CRISPR–Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing

(2019) Benjamin P. Kleinstiver et al.   NATURE BIOTECHNOLOGY

CasX enzymes comprise a distinct family of RNA-guided genome editors

(2019) Jun-Jie Liu et al.   NATURE

hESC-Derived Thalamic Organoids Form Reciprocal Projections When Fused with Cortical Organoids

(2019) Yangfei Xiang et al.   Cell Stem Cell

Defining the Genetic, Genomic, Cellular, and Diagnostic Architectures of Psychiatric Disorders

(2019) Patrick F. Sullivan et al.   CELL

Overexpression of NEUROG2 and NEUROG1 in human embryonic stem cells produces a network of excitatory and inhibitory neurons

(2019) Congyi Lu et al.   FASEB JOURNAL

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

Multiplexed detection of proteins, transcriptomes, clonotypes and CRISPR perturbations in single cells

(2019) Eleni P. Mimitou et al.   NATURE METHODS

Individual brain organoids reproducibly form cell diversity of the human cerebral cortex

(2019) Silvia Velasco et al.   NATURE

Neuroligin-4 Regulates Excitatory Synaptic Transmission in Human Neurons

(2019) Samuele G. Marro et al.   NEURON

CRISPR-based functional evaluation of schizophrenia risk variants

(2019) Prashanth Rajarajan et al.   SCHIZOPHRENIA RESEARCH

Synergistic effects of common schizophrenia risk variants

(2019) Nadine Schrode et al.   NATURE GENETICS

CRISPR Interference-Based Platform for Multimodal Genetic Screens in Human iPSC-Derived Neurons

(2019) Ruilin Tian et al.   NEURON

Identification and Massively Parallel Characterization of Regulatory Elements Driving Neural Induction

(2019) Fumitaka Inoue et al.   Cell Stem Cell

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Engineering of human brain organoids with a functional vascular-like system

(2019) Bilal Cakir et al.   NATURE METHODS

The great hairball gambit

(2019) Jonathan Flint et al.   PLoS Genetics

Landscape of Conditional eQTL in Dorsolateral Prefrontal Cortex and Co-localization with Schizophrenia GWAS

(2018) Amanda Dobbyn et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

(2018) Silvana Konermann et al.   CELL

Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene

(2018) X. Shawn Liu et al.   CELL

Dissecting the Functional Consequences of De Novo DNA Methylation Dynamics in Human Motor Neuron Differentiation and Physiology

(2018) Michael J. Ziller et al.   Cell Stem Cell

Genome and epigenome engineering CRISPR toolkit for in vivo modulation of cis -regulatory interactions and gene expression in the chicken embryo

(2018) Ruth M. Williams et al.   DEVELOPMENT

Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells

(2018) Miaojin Zhou et al.   HUMAN GENE THERAPY

An in vivo model of functional and vascularized human brain organoids

(2018) Abed AlFatah Mansour et al.   NATURE BIOTECHNOLOGY

Genetic identification of brain cell types underlying schizophrenia

(2018) Nathan G. Skene et al.   NATURE GENETICS

Heritability enrichment of specifically expressed genes identifies disease-relevant tissues and cell types

(2018) Hilary K. Finucane et al.   NATURE GENETICS

The CRISPR tool kit for genome editing and beyond

(2018) Mazhar Adli   Nature Communications

Prediction of response to drug therapy in psychiatric disorders

(2018) Shani Stern et al.   Open Biology

Sodium valproate rescues expression of TRANK1 in iPSC-derived neural cells that carry a genetic variant associated with serious mental illness

(2018) Xueying Jiang et al.   MOLECULAR PSYCHIATRY

Rapid and efficient induction of functional astrocytes from human pluripotent stem cells

(2018) Isaac Canals et al.   NATURE METHODS

CRISPR Activation Screens Systematically Identify Factors that Drive Neuronal Fate and Reprogramming

(2018) Yanxia Liu et al.   Cell Stem Cell

Reliability of human cortical organoid generation

(2018) Se-Jin Yoon et al.   NATURE METHODS

Topological defects control collective dynamics in neural progenitor cell cultures

(2017) Kyogo Kawaguchi et al.   NATURE

RNA targeting with CRISPR–Cas13

(2017) Omar O. Abudayyeh et al.   NATURE

Assembly of functionally integrated human forebrain spheroids

(2017) Fikri Birey et al.   NATURE

Cell diversity and network dynamics in photosensitive human brain organoids

(2017) Giorgia Quadrato et al.   NATURE

CRISPR–Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome

(2017) Tyler S Klann et al.   NATURE BIOTECHNOLOGY

Rare coding variants in PLCG2, ABI3, and TREM2 implicate microglial-mediated innate immunity in Alzheimer's disease

(2017) Rebecca Sims et al.   NATURE GENETICS

CRISPR–Cas9-based photoactivatable transcription systems to induce neuronal differentiation

(2017) Yuta Nihongaki et al.   NATURE METHODS

Inducible and multiplex gene regulation using CRISPR–Cpf1-based transcription factors

(2017) Y Esther Tak et al.   NATURE METHODS

Generation of pure GABAergic neurons by transcription factor programming

(2017) Nan Yang et al.   NATURE METHODS

Fused cerebral organoids model interactions between brain regions

(2017) Joshua A Bagley et al.   NATURE METHODS

Pooled CRISPR screening with single-cell transcriptome readout

(2017) Paul Datlinger et al.   NATURE METHODS

Opportunities and challenges in phenotypic drug discovery: an industry perspective

(2017) John G. Moffat et al.   NATURE REVIEWS DRUG DISCOVERY

iPSC-Derived Human Microglia-like Cells to Study Neurological Diseases

(2017) Edsel M. Abud et al.   NEURON

Modeling neurodegenerative diseases with patient-derived induced pluripotent cells: Possibilities and challenges

(2017) Anna Poon et al.   New Biotechnology

Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors

(2017) Marc Ehrlich et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

Derivation of Functional Human Astrocytes from Cerebral Organoids

(2017) Rômulo Sperduto Dezonne et al.   Scientific Reports

Evaluating Synthetic Activation and Repression of Neuropsychiatric-Related Genes in hiPSC-Derived NPCs, Neurons, and Astrocytes

(2017) Seok-Man Ho et al.   Stem Cell Reports

Simple Derivation of Spinal Motor Neurons from ESCs/iPSCs Using Sendai Virus Vectors

(2017) Kazuya Goto et al.   Molecular Therapy-Methods & Clinical Development

Multiplex gene regulation by CRISPR-ddCpf1

(2017) Xiaochun Zhang et al.   Cell Discovery

FINEMAP: efficient variable selection using summary data from genome-wide association studies

(2016) Christian Benner et al.   BIOINFORMATICS

Perturb-Seq: Dissecting Molecular Circuits with Scalable Single-Cell RNA Profiling of Pooled Genetic Screens

(2016) Atray Dixit et al.   CELL

A Multiplexed Single-Cell CRISPR Screening Platform Enables Systematic Dissection of the Unfolded Protein Response

(2016) Britt Adamson et al.   CELL

Brain-Region-Specific Organoids Using Mini-bioreactors for Modeling ZIKV Exposure

(2016) Xuyu Qian et al.   CELL

Editing DNA Methylation in the Mammalian Genome

(2016) X. Shawn Liu et al.   CELL

Genetic variants associated with response to lithium treatment in bipolar disorder: a genome-wide association study

(2016) Liping Hou et al.   LANCET

Advancing drug discovery for neuropsychiatric disorders using patient-specific stem cell models

(2016) Stephen J. Haggarty et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Altered proliferation and networks in neural cells derived from idiopathic autistic individuals

(2016) M C Marchetto et al.   MOLECULAR PSYCHIATRY

Schizophrenia risk from complex variation of complement component 4

(2016) Aswin Sekar et al.   NATURE

Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array

(2016) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Spatial genome organization and cognition

(2016) Prashanth Rajarajan et al.   NATURE REVIEWS NEUROSCIENCE

Dysregulation of miRNA-9 in a Subset of Schizophrenia Patient-Derived Neural Progenitor Cells

(2016) Aaron Topol et al.   Cell Reports

Sustained synchronized neuronal network activity in a human astrocyte co-culture system

(2016) Jacobine Kuijlaars et al.   Scientific Reports

Generation of functional human serotonergic neurons from fibroblasts

(2015) K C Vadodaria et al.   MOLECULAR PSYCHIATRY

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

(2015) Silvana Konermann et al.   NATURE

Differential responses to lithium in hyperexcitable neurons from patients with bipolar disorder

(2015) Jerome Mertens et al.   NATURE

Highly efficient Cas9-mediated transcriptional programming

(2015) Alejandro Chavez et al.   NATURE METHODS

Functional cortical neurons and astrocytes from human pluripotent stem cells in 3D culture

(2015) Anca M Paşca et al.   NATURE METHODS

Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements

(2015) Pratiksha I Thakore et al.   NATURE METHODS

A Protein-Tagging System for Signal Amplification in Gene Expression and Fluorescence Imaging

(2014) Marvin E. Tanenbaum et al.   CELL

Phenotypic differences in hiPSC NPCs derived from patients with schizophrenia

(2014) K Brennand et al.   MOLECULAR PSYCHIATRY

A three-dimensional human neural cell culture model of Alzheimer’s disease

(2014) Se Hoon Choi et al.   NATURE

Bayesian Test for Colocalisation between Pairs of Genetic Association Studies Using Summary Statistics

(2014) Claudia Giambartolomei et al.   PLoS Genetics

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

(2013) Lei S. Qi et al.   CELL

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

Functional Maturation of hPSC-Derived Forebrain Interneurons Requires an Extended Timeline and Mimics Human Neural Development

(2013) Cory R. Nicholas et al.   Cell Stem Cell

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Rapid Single-Step Induction of Functional Neurons from Human Pluripotent Stem Cells

(2013) Yingsha Zhang et al.   NEURON

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Rapid Generation of Functional Dopaminergic Neurons From Human Induced Pluripotent Stem Cells Through a Single-Step Procedure Using Cell Lineage Transcription Factors

(2013) Ilda Theka et al.   Stem Cells Translational Medicine

Modeling human cortical development in vitro using induced pluripotent stem cells

(2012) J. Mariani et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Modelling schizophrenia using human induced pluripotent stem cells

(2011) Kristen J. Brennand et al.   NATURE