A novel mRNA-based therapy that induces corneal endothelium regeneration to restore vision loss
Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells.
Lipid nanoparticles for selective mRNA delivery to the placenta to treat pre-eclampsia, FGR, and other pregnancy-related disorders.
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac
Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment
A split enzyme complex enabling precise gene editing
CRISPR-Cas12a allowing for simultaneous editing of multiple genes in mammalian cells
Duchenne Muscular Dystrophy (DMD) therapy deletes let-7c miRNA binding sites using CRISPR-Cas9 based genome editing and AAV based delivery.
Gene therapy targeting disposal of toxic intracellular lipid debris and lipofuscin in wet age related macular degeneration.