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.
High-throughput in vivo screening method to identify lipid nanoparticle (LNP) formulations for local and systemic delivery for mRNA vaccines and cancer immunotherapies.
siRNA-LNP knock-down of TAK1 delivered intranasally and to macrophages specifically prevents inflammation-induced pathology in a mouse model
Targeted lipid nanoparticles (LNPs) that can deliver mRNA-based gene editing therapeutics to hematopoietic stem cells (HSCs) in utero
Lipid nanoparticles (LNPs) with reversibly attached branched lipid tails for the delivery of mRNA for gene therapy.
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.