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Nat Neurosci ; 23(12): 1629-1636, 2020 12.
Artigo em Inglês | MEDLINE | ID: mdl-32807948

RESUMO

Recent success in identifying gene-regulatory elements in the context of recombinant adeno-associated virus vectors has enabled cell-type-restricted gene expression. However, within the cerebral cortex these tools are largely limited to broad classes of neurons. To overcome this limitation, we developed a strategy that led to the identification of multiple new enhancers to target functionally distinct neuronal subtypes. By investigating the regulatory landscape of the disease gene Scn1a, we discovered enhancers selective for parvalbumin (PV) and vasoactive intestinal peptide-expressing interneurons. Demonstrating the functional utility of these elements, we show that the PV-specific enhancer allowed for the selective targeting and manipulation of these neurons across vertebrate species, including humans. Finally, we demonstrate that our selection method is generalizable and characterizes additional PV-specific enhancers with exquisite specificity within distinct brain regions. Altogether, these viral tools can be used for cell-type-specific circuit manipulation and hold considerable promise for use in therapeutic interventions.


Assuntos
Dependovirus/genética , Vetores Genéticos/genética , Interneurônios/fisiologia , Animais , Callithrix , Córtex Cerebral/citologia , Feminino , Humanos , Macaca mulatta , Camundongos , Camundongos Endogâmicos C57BL , Canal de Sódio Disparado por Voltagem NAV1.1/genética , Neurônios , Parvalbuminas/fisiologia , Ratos , Ratos Sprague-Dawley , Especificidade da Espécie , Peptídeo Intestinal Vasoativo/fisiologia
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