The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects.
Lung-targeting lipid nanoparticles allowing organ specific mRNA delivery for gene therapy applications.
Lipid nanoparticles (LNPs) with reversibly attached branched lipid tails for the delivery of mRNA for gene therapy.
Approaches and Design Techniques to Alleviate Coagulation-Related Acute Side Effects of Lung-Targeted Nanocarriers
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
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