Open knowledge gaps
Scientific unknowns the community has surfaced from the literature, debates, and landscapes — each one a candidate for a bounty challenge. Pick a gap, fund it, and the substrate runs the resolution.
Showing neurotherapeutics gaps, sorted by Newest.
How can cell-autonomous gene therapy treatments be designed to address epilepsy's complex pathogenic mechanisms?
neurotherapeutics openThe abstract suggests cell-autonomous treatments may be crucial for addressing epilepsy gene therapy challenges, but doesn't explain how to achieve cell-specific targeting given epilepsy's complex, multi-cellular pathog…
gap-pubmed-20260411-072417-b40dfc53How can gene therapy expression be precisely controlled on-demand for the delicate excitatory/inhibitory balance in epilepsy?
neurotherapeutics openThe abstract identifies expression controllability as a major challenge, noting that reconfiguring excitatory and inhibitory properties in epilepsy is a delicate process requiring on-demand control. Current gene therapy…
gap-pubmed-20260411-072417-76e9c437How does fosgonimeton cross the blood-brain barrier to achieve CNS effects?
neurotherapeutics resolvedThe study demonstrates clear CNS efficacy in vivo but doesn't address the pharmacokinetic mechanism by which this small molecule HGF modulator reaches brain tissue. This gap is critical for understanding dosing strategi…
gap-pubmed-20260410-185235-bb913ca8What mechanisms enable neuropeptide-loaded MSC-EVs to cross the blood-brain barrier via intranasal delivery?
neurotherapeutics openThe study demonstrates BBB crossing but doesn't elucidate the transport mechanisms involved. This knowledge gap limits optimization of delivery efficiency and understanding of EV biodistribution in the CNS. Gap type: un…
gap-pubmed-20260410-181230-4cdd6f61Why do therapeutic effects persist for 6 months after single ds-cRNA administration?
neurotherapeutics openThe sustained therapeutic benefit lasting at least 6 months from a single treatment is remarkable but mechanistically unexplained. Understanding whether this reflects circular RNA stability, epigenetic changes, or other…
gap-pubmed-20260410-174005-2f2fc1abWhat determines the specificity and efficiency of mitochondrial uptake by different brain cell types in vivo?
neurotherapeutics openWhile the study shows therapeutic effects after intravenous delivery, it's unclear how transplanted mitochondria cross the blood-brain barrier and selectively target affected brain regions. This knowledge gap is essenti…
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