What they found
Systematic review and meta-analysis of 9 preclinical studies on FUS-mediated gene therapy for orthotopic glioblastoma. Tested viral, non-viral, nanoparticle, and exosome-based vectors delivering therapeutic genes (MDA-7/IL-24, HSV-TK, CRISPR/Cas9). FUS significantly enhanced gene expression (pooled effect size 6.34, 95% CI 2.21-18.18), reduced tumor volume (pooled effect size 4.03, 95% CI 1.46-11.12), and improved survival (HR 1.33, 95% CI 1.13-1.56).
Lateral connection
The 6.34x enhancement of gene expression via FUS is a quantitative benchmark applicable to inner ear gene therapy. If FUS could similarly enhance AAV transduction of outer hair cells through the blood-labyrinth barrier, the required AAV dose for mini-STRC delivery would drop proportionally — potentially solving both the dosing and immunogenicity challenges. The meta-analysis across multiple vector types (viral, non-viral, exosome) suggests the FUS enhancement is vector-agnostic, meaning it would likely work with AAV-mini-STRC.
Hypothesis suggested
FUS applied to the cochlea during systemic or local AAV-mini-STRC administration could enhance outer hair cell transduction 3-6x compared to passive delivery, achieving therapeutic stereocilin levels with lower vector doses.
What could be computed
Dose-response modeling: given known AAV transduction efficiencies in cochlear studies, what minimum FUS-enhancement factor is needed to achieve >50% OHC transduction with clinically feasible AAV doses? Sensitivity analysis across reported enhancement ranges (2.2x to 18.2x from the meta-analysis CI).
Links
Connections
[source]auto-indexed 2026-04-20 by strc-lit-watch