Technology allowing efficient formation, delivery, isolation, and enrichment of single-copy Human Artificial Chromosome (HAC) for use in research, plant production, gene and cell therapy and more.
Ionizable LNPs with a rigid backbone of piperazine (PIP) linked to bisphosphonates (BPs) for the targeted delivery of mRNA to the bone.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
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.
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.