Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
Liver-targeted small RNA interference (RNAi) therapy to treat nonalcoholic fatty liver diseases (NAFLD), including nonalcoholic steatohepatitis (NASH).
A method of treating Fragile X syndrome by correcting the abnormal DNA clusters causing the characteristic symptoms.
A novel mRNA-based therapy that induces corneal endothelium regeneration to restore vision loss
Lipid nanoparticles for selective mRNA delivery to the placenta to treat pre-eclampsia, FGR, and other pregnancy-related disorders.
Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells.
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.
Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment
CRISPR-Cas12a allowing for simultaneous editing of multiple genes in mammalian cells