Independent disease-relevant outcomes are missing.
The current response substrate contains observed CRISPRi perturbations in HepG2, plus limited muscle-context and correction references. None is an independent candidate perturbation in a DMD muscle model.
- Observed same-context responses exist for 21 candidates.
- Only 9 candidates were represented in the external myoblast screen.
- No released candidate has an independent DMD muscle perturbation outcome.