Abstract: Artificial expression constructs for modulating gene expression in GABAergic neurons and astrocytes are described. The artificial expression constructs can be used to express SLC6A1 for the treatment of SLC6A1-associated disorders, among other uses.

Abstract: Artificial expression constructs for modulating gene expression in targeted central nervous system cell types are described. The artificial expression constructs can be used to express synthetic genes or modify gene expression in chandelier cells. Chandelier cells are a subtype of GABAergic interneurons that that have been implicated in disorders such as epilepsy and schizophrenia.

Abstract: Artificial expression constructs for modulating gene expression in striatal neurons are described. The artificial expression constructs can be used to express heterologous genes in striatal neurons including in striatal medium spiny neuron-pan, striatal medium spiny neuron-indirect pathway, striatal medium spiny neuron-direct pathway, striatal interneuron-cholinergic, and Drd3+ medium spiny neurons in olfactory tubercle. The artificial expression constructs can be used for many purposes, including to research and treat movement disorders such as Parkinson's disease and Huntington's disease.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in inhibitory neocortical GABAergic neurons including somatostatin GABAergic neurons, parvalbumin GABAergic neurons, vasointestinal peptide GABAergic neurons, Lamp5 GABAergic neurons, and in some instances astrocytes.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. Particularly, the artificial expression constructs can be used to selectively express synthetic genes and/or modify gene expression in neocortical glutamatergic layer 5 neurons, such as glutamatergic layer 5 extratelencephalic-projecting (L5 ET) neurons.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in astrocytes, oligodendrocytes, microglia, pericytes, SMC, or endothelial cells.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in GABAergic neurons generally; and/or GABAergic neuron cell types such as lysosomal associated membrane protein 5 (Lamp5) neurons; vasoactive intestinal polypeptide-expressing (Vip) neurons; somatostatin (Sst) neurons; and/or parvalbumin (Pvalb) neuron cell types. Certain artificial expression constructs additionally drive selective gene expression in Layer 4 and/or layer 5 intratelencephalic (IT) neurons, deep cerebellar nuclear neurons or cerebellar Purkinje cells.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in excitatory cortical neurons, such as primarily within cortical layers 2/3, 4, 5, and 6 and including those with extratelencephalic (ET) projections, intratelencephalic (IT) projections, and pyramidal tract (PT) projections, among others.

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in GABAergic interneurons.

Abstract: Selectively providing voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function to inhibitory neurons is described. Provided voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function in inhibitory neurons can be used to treat disorders such as epilepsy, and more particularly, Dravet Syndrome.

Abstract: The disclosure reports on the identification and isolation of adult mouse lateral ventricle subventricular zone (SVZ) neurosphere initiating cells (NICs) by flow cytometry on the basis of GlastmidEGFRhighPlexinB2highCD24?/lowO4/PSA-NCAM?/lowTer-119/CD45? markers (GEPCOT cells). These cells are highly mitotic and short-lived in vivo based on fate-mapping with Ascl1CreERT2 and Dlx1CreERT2. In contrast, pre-GEPCOT cells were quiescent, expressed higher Glast, and lower EGFR and PlexinB2. Pre-GEP-COT cells could not form neurospheres but expressed the stem cell markers Glast-CreERT, GFAP-CreERT2, Sox2CreERT2, and Gli1C-reERT2 and were long-lived in vivo. While GEPCOT NICs were ablated by temozolomide, pre-GEPCOT cells survived and repopulated the SVZ. Conditional deletion of the Bmi-1 polycomb protein depleted pre-GEPCOT and GEPCOT cells, though pre-GEPCOT cells were more dependent upon Bmi-1 for p16Ink4a repression.