miRNA-Mediated Transgene Expression System for Improved Gene Therapies and Research Reagent Applications
A highly specific and tailorable transgene expression systems compatible with conventional gene therapy platforms
Screening Method and Gene Therapy Approach for Treating Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia (ARVC/D)
An innovative and efficacious treatment targeting the genetic drivers of arrhythmogenic right ventricular cardiomyopathy/dysplasia (ARVC/D).
Viral Vector Expression of Gabrb1b for the Treatment of Perceptual Deficits
Innovative and targeted approaches to treat perceptual deficits associated with reduced GABA receptor-mediated inhibitory postsynaptic potentials.