🎯 Drug Targets

GABRA1 mediates inhibitory neurotransmission through GABA-A receptor subunit composition, modulating neuronal excitability and synaptic signaling. In neurodegeneration contexts, alterations in GABRA1 expression or function can contribute to neuronal hyperexcitability, oxidative stress, and potentially accelerated cellular damage mechanisms associated with progressive neurological disorders.

Upregulation through PCSK9 inhibition or direct receptor modulation

Small molecule blocker of T-type calcium channel activity

Small molecule agonist or antagonist modulating dopamine signaling

Small molecule inhibitor of mutant IDH2 enzyme activity

Selective P2X7 receptor antagonists blocking ATP-gated ion channel

Small molecule inhibitor of anti-apoptotic protein function

RAB7A inhibitors or modulators would block or enhance GTPase-mediated late endosome and lysosome trafficking, disrupting autophagy flux and lysosomal degradation pathways. This can either impair pathogenic protein clearance (in certain cancers) or restore lysosomal function (in storage disorders and neurodegeneration).

Small molecule agonists that activate G-protein coupled receptor signaling

Prioritized from 1 SciDEX hypotheses, including: Purinergic Signaling Polarization Control