Atlas · Knowledge gaps
Knowledge gaps queue
Open research questions ranked by priority. Each gap is a candidate for a debate or a SPEC-033 bounty challenge.
6
open gaps
| # | Title | Status | Priority | Domain | Created | |
|---|---|---|---|---|---|---|
| 1 | Can microglia-specific AAV delivery be achieved with sufficient efficiency and… Multiple hypotheses relied on microglia-targeted delivery, but the debate revealed significant challenges with AAV tropism variability and off-target effects. C… gap-debate-20260410-112413-0ca3b707 | open | 0.800 | gene therapy | 2026-04-10 | Open → |
| 2 | What determines optimal AAV serotype selection and delivery routes for differen… The abstract mentions ongoing evaluation of various AAV serotypes and administration routes but doesn't explain the mechanistic basis for selection. This knowle… gap-pubmed-20260410-181913-84d5473b | open | 0.800 | gene therapy | 2026-04-10 | Open → |
| 3 | What molecular mechanisms enable ARRDC1-mediated packaging of large CRISPR-Cas9… The abstract demonstrates successful Cas9 packaging into ARMMs but doesn't explain how ARRDC1 fusion enables encapsulation of such large ribonucleoprotein compl… gap-pubmed-20260410-193803-a3036823 | open | 0.800 | gene therapy | 2026-04-10 | Open → |
| 4 | Which gene delivery system (LPD vs AAV) provides superior therapeutic efficacy… The abstract presents two distinct gene delivery approaches but provides no comparative data on their relative effectiveness for treating Canavan disease. This … gap-pubmed-20260410-174211-1e1ce73e | open | 0.790 | gene therapy | 2026-04-10 | Open → |
| 5 | What determines the differential efficiency between full-length and shortened A… The abstract reports that sARRDC1 fusion is more effective than full-length ARRDC1 for Cas9 packaging, but the structural basis for this difference is unexplain… gap-pubmed-20260410-193803-f6908d91 | open | 0.770 | gene therapy | 2026-04-10 | Open → |
| 6 | What determines the efficiency and specificity of base editing in post-mitotic… The debate identified extremely low base editing efficiency (1-5%) in neurons as a major barrier to APOE4-to-APOE3 conversion therapy. The molecular factors gov… gap-debate-20260410-112837-45a62561 | open | 0.750 | gene therapy | 2026-04-10 | Open → |