Human Neuropsychiatric Disease Modeling using Conditional Deletion Reveals Synaptic Transmission Defects Caused by Heterozygous Mutations in NRXN1
Human Neuropsychiatric Disease Modeling using Conditional Deletion Reveals Synaptic Transmission Defects Caused by Heterozygous Mutations in NRXN1
Heterozygous mutations of the NRXN1 gene, which encodes the presynaptic cell-adhesion molecule neurexin-1, were repeatedly associated with autism and schizophrenia. However, diverse clinical presentations of NRXN1 mutations in patients raise the question of whether heterozygous NRXN1 mutations alone directly impair synaptic function. To address this question under conditions that precisely control for genetic background, we generated human ESCs with different heterozygous conditional NRXN1 mutations and analyzed two different types of isogenic control and NRXN1 mutant neurons derived from these ESCs. Both heterozygous NRXN1 mutations selectively impaired neurotransmitter release in human neurons without changing neuronal differentiation or synapse formation. Moreover, both NRXN1 mutations increased the levels of CASK, a critical synaptic scaffolding protein that binds to neurexin-1. Our results show that, unexpectedly, heterozygous inactivation of NRXN1 directly impairs synaptic function in human neurons, and they illustrate the value of this conditional deletion approach for studying the functional effects of disease-associated mutations.
- Howard Hughes Medical Institute United States
- University of California, San Francisco United States
- Institute for Stem Cell Biology and Regenerative Medicine United States
- Stanford University School of Medicine United States
- Stanford University United States
Neuronal, Autism, Human Embryonic Stem Cells, Regenerative Medicine, Synaptic Transmission, Medical and Health Sciences, neurexin, Gene Knockout Techniques, Models, synapse, Enzyme Stability, Neural Cell Adhesion Molecules, Neurons, Neurotransmitter Agents, Mental Disorders, Miniature Postsynaptic Potentials, Cell Differentiation, Biological Sciences, Mental Health, Phenotype, Neurological, synaptic cell adhesion, Gene Targeting, Molecular Medicine, Synaptic Vesicles, Heterozygote, Intellectual and Developmental Disabilities (IDD), Cell Adhesion Molecules, Neuronal, Molecular Sequence Data, autism, Nerve Tissue Proteins, Models, Biological, iN cells, Genetics, Humans, Amino Acid Sequence, Stem Cell Research - Embryonic - Human, human neurons, Calcium-Binding Proteins, Cell Membrane, Neurosciences, Cell Biology, Stem Cell Research, Biological, Brain Disorders, schizophrenia, Synapses, Mutation, Schizophrenia, Cell Adhesion Molecules, Guanylate Kinases, Developmental Biology
Neuronal, Autism, Human Embryonic Stem Cells, Regenerative Medicine, Synaptic Transmission, Medical and Health Sciences, neurexin, Gene Knockout Techniques, Models, synapse, Enzyme Stability, Neural Cell Adhesion Molecules, Neurons, Neurotransmitter Agents, Mental Disorders, Miniature Postsynaptic Potentials, Cell Differentiation, Biological Sciences, Mental Health, Phenotype, Neurological, synaptic cell adhesion, Gene Targeting, Molecular Medicine, Synaptic Vesicles, Heterozygote, Intellectual and Developmental Disabilities (IDD), Cell Adhesion Molecules, Neuronal, Molecular Sequence Data, autism, Nerve Tissue Proteins, Models, Biological, iN cells, Genetics, Humans, Amino Acid Sequence, Stem Cell Research - Embryonic - Human, human neurons, Calcium-Binding Proteins, Cell Membrane, Neurosciences, Cell Biology, Stem Cell Research, Biological, Brain Disorders, schizophrenia, Synapses, Mutation, Schizophrenia, Cell Adhesion Molecules, Guanylate Kinases, Developmental Biology
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