IndraLab

Statements

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"In addition, increasing GABA signalling by application of the positive allosteric GABA A receptor modulator clonazepam was sufficient to rescue the abnormal social behaviour in Scn1a +/- mice 181."
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"Significantly, the KT-182-potentiation of tonic GABA R-mediated currents in GCs was greater in Scn1a hippocampal slices (PND35–45) compared to WT slices, and was absent in Abhd6 (Fig. 4E)."
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"As an illustration, Han et al. (2012) showed that enhancing GABA-mediated signaling (with clonazepam) in Scn1a +/− mice rescued autistic-like behaviors and cognitive comorbidities, demonstrating that these deficits are at least in part due to reduced GABAergic tone.Overall, our results highlight the direct role of Nav1.1 in IN fast spiking activity and the impact their dysregulation can have at the network and behavioral levels."
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"ETX101 is a recombinant adeno-associated viral (AAV) vector containing a GABA regulatory element designed to upregulate SCN1A (encoding the voltage-gated sodium channel NaV1.1) and restore interneuron function in patients with Dravet Syndrome.Similar to ETX101, STK-001 is an antisense oligonucleotide designed to enhance the generation of wild-type SCN1A mRNA transcripts to restore inhibitory function in Dravet Syndrome (Bialer et al., 2020; Wengert et al., 2022)."
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