Computational hypotheses for accelerating STRC gene therapy. These require experimental validation.
A truncated STRC construct (3984 bp) that fits within the AAV packaging limit. Leverages AlphaFold pLDDT to identify dispensable disordered N-terminal domains.
Precision correction of c.4976A>C using PE3 or PE3b pegRNA strategy without double-strand breaks.
Mechanosensitive channel activation via focused ultrasound. ODE model of calcium signaling in OHC stereocilia.
Non-invasive cochlear gene delivery via sonoporation + LNP. ODE model shows 78% transduction efficiency under optimized parameters.
Poisson stochastic model quantifying the probability cost of dual-vector co-transduction vs single-vector strategy.
Coulomb energetics explain why E1659A is pathogenic despite intact structure. 8.62 kcal/mol destabilization of adhesion interface.
NAb kinetics, seroprevalence barriers, and delivery vehicle comparison for cochlear gene therapy re-dosing strategies.