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Article
Evaluation of AAV Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy
Nosebleeds and intracranial hemorrhage from brain arteriovenous malformations (bAVMs) are among the most devastating symptoms of patients with hereditary hemorrhagic telangiectasis (HHT). All available managem...
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Article
Open AccessTowards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
Our understanding of neurological diseases has been tremendously enhanced over the past decade by the application of new technologies. Genome-wide association studies have highlighted glial cells as important ...
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Article
Open AccessReactive astrocytes transduce inflammation in a blood-brain barrier model through a TNF-STAT3 signaling axis and secretion of alpha 1-antichymotrypsin
Astrocytes are critical components of the neurovascular unit that support blood-brain barrier (BBB) function. Pathological transformation of astrocytes to reactive states can be protective or harmful to BBB fu...
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Article
CRISPRi screens in human iPSC-derived astrocytes elucidate regulators of distinct inflammatory reactive states
Astrocytes become reactive in response to insults to the central nervous system by adopting context-specific cellular signatures and outputs, but a systematic understanding of the underlying molecular mechanis...
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Article
Open AccessA CRISPRi/a platform in human iPSC-derived microglia uncovers regulators of disease states
Microglia are emerging as key drivers of neurological diseases. However, we lack a systematic understanding of the underlying mechanisms. Here, we present a screening platform to systematically elucidate funct...
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Article
Genome-wide CRISPRi/a screens in human neurons link lysosomal failure to ferroptosis
Single-cell transcriptomics provide a systematic map of gene expression in different human cell types. The next challenge is to systematically understand cell-type-specific gene function. The integration of CR...
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Article
Patterns of neuronal Rhes as a novel hallmark of tauopathies
The farnesyltransferase inhibitor, Lonafarnib, reduces tau inclusions and associated atrophy in familial tauopathy models through activation of autophagy, mediated by the inhibition of farnesylation of the Ras...
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Article
Molecular characterization of selectively vulnerable neurons in Alzheimer’s disease
Alzheimer’s disease (AD) is characterized by the selective vulnerability of specific neuronal populations, the molecular signatures of which are largely unknown. To identify and characterize selectively vulner...
