Monoclonal antibody against Annexin A1 blocks Fusobacterium nucleatum invasion, offering targeted prevention and treatment of Fn-related cancers.
Biomarker predicts AIHA onset; apyrase treatment prophylactically/therapeutically mitigates AIHA in preclinical models.
Lysosomal phospholipase A2 enables BMP biosynthesis modulation to study and treat BMP-related disorders, including Niemann-Pick disease.
Retinoid pathway inverse agonists reprogram ACC cells to non-tumorigenic states, enabling targeted differentiation and death.
ATP7B knockout mouse models Wilson disease, enabling in vivo study of copper accumulation, liver cirrhosis-like pathology, and therapy screening.
Neuron-specific OCRL1 mutant study reveals brain-focused function; enables Lowe syndrome models, diagnostics, and targeted therapies.
Physiological, inducible CPVT mouse model with RYR2 mutation; enables in vivo study of AF/VT and antiarrhythmic drug development.
RNA-based therapy using antisense long non-coding RNA to modulate SLC2A1/Glut1 expression for Glut1 deficiency treatment.