Recombinant AAV vectors deliver IRF2BPL cDNA with optimized promoters to restore functional protein expression in IRF2BPL disorders.
A biomimetic weathering enzyme thins glass uniformly to 20 microns while avoiding fluoride toxicity, roughness, and complex cleaning.
An ultra-miniaturized TnpB and reRNA system enables target gene regulation within AAV cargo limits while avoiding DNA cleavage damage.
Cell wall polysaccharide-degrading enzymes smooth plant-based milk texture and improve taste without complex micronization or homogenization.
AAV delivery of truncated ATP7B removes liver copper and limits reaccumulation while improving manufacturing yield over wild-type constructs.