Massively parallel techniques for cataloguing the regulome of the human brain
Complex brain disorders are highly heritable and arise from a complex polygenic risk
architecture. Many disease-associated loci are found in non-coding regions that house …
architecture. Many disease-associated loci are found in non-coding regions that house …
Modeling the complex genetic architectures of brain disease
The genetic architecture of each individual comprises common and rare variants that, acting
alone and in combination, confer risk of disease. The cell-type-specific and/or context …
alone and in combination, confer risk of disease. The cell-type-specific and/or context …
CRISPR-based functional genomics for neurological disease
M Kampmann - Nature Reviews Neurology, 2020 - nature.com
Neurodegenerative, neurodevelopmental and neuropsychiatric disorders are among the
greatest public health challenges, as many lack disease-modifying treatments. A major …
greatest public health challenges, as many lack disease-modifying treatments. A major …
Evaluating synthetic activation and repression of neuropsychiatric-related genes in hiPSC-derived NPCs, neurons, and astrocytes
SM Ho, BJ Hartley, E Flaherty, P Rajarajan, R Abdelaal… - Stem cell reports, 2017 - cell.com
Modulation of transcription, either synthetic activation or repression, via dCas9-fusion
proteins is a relatively new methodology with the potential to facilitate high-throughput up-or …
proteins is a relatively new methodology with the potential to facilitate high-throughput up-or …
CRISPR interference-based platform for multimodal genetic screens in human iPSC-derived neurons
CRISPR/Cas9-based functional genomics have transformed our ability to elucidate
mammalian cell biology. However, most previous CRISPR-based screens were conducted …
mammalian cell biology. However, most previous CRISPR-based screens were conducted …
Population-scale single-cell RNA-seq profiling across dopaminergic neuron differentiation
Studying the function of common genetic variants in primary human tissues and during
development is challenging. To address this, we use an efficient multiplexing strategy to …
development is challenging. To address this, we use an efficient multiplexing strategy to …
Application of CRISPR/Cas9 to the study of brain development and neuropsychiatric disease
SK Powell, J Gregory, S Akbarian… - Molecular and Cellular …, 2017 - Elsevier
CRISPR/Cas9 technology has transformed our ability to manipulate the genome and
epigenome, from efficient genomic editing to targeted localization of effectors to specific loci …
epigenome, from efficient genomic editing to targeted localization of effectors to specific loci …
[HTML][HTML] Genome-scale CRISPR screens identify human pluripotency-specific genes
Human pluripotent stem cells (hPSCs) generate a variety of disease-relevant cells that can
be used to improve the translation of preclinical research. Despite the potential of hPSCs …
be used to improve the translation of preclinical research. Despite the potential of hPSCs …
A CRISPRi/a platform in human iPSC-derived microglia uncovers regulators of disease states
NM Dräger, SM Sattler, CTL Huang, OM Teter… - Nature …, 2022 - nature.com
Microglia are emerging as key drivers of neurological diseases. However, we lack a
systematic understanding of the underlying mechanisms. Here, we present a screening …
systematic understanding of the underlying mechanisms. Here, we present a screening …
Human pluripotent stem cell–derived models and drug screening in CNS precision medicine
MC Silva, SJ Haggarty - Annals of the New York Academy of …, 2020 - Wiley Online Library
Abstract Development of effective therapeutics for neurological disorders has historically
been challenging partly because of lack of accurate model systems in which to investigate …
been challenging partly because of lack of accurate model systems in which to investigate …