We develop technologies for delivering nucleic acids to cells ex vivo and in vivo.
Our nucleic-acid delivery technologies enable efficient ex vivo delivery of mRNA encoding gene-editing proteins and reprogramming factors, including to primary cells, insertion of exogenous sequences into genomic safe-harbor loci, and efficient in vivo delivery of mRNA to the brain, eye, skin, and lung.
ToRNAdo™ Nucleic-Acid Delivery System
Delivery systems can be used to enhance the uptake of nucleic acids by cells. Conventional delivery systems often suffer from endosomal entrapment and toxicity, which can limit their therapeutic use. Our scientists developed a novel chemical substance that is exceptionally effective at delivering nucleic acids, including mRNA, to cells both ex vivo and in vivo. […]
Insertion of Sequences into Safe-Harbor Loci
Safe-harbor loci are regions of the genome that can be disrupted in a wide variety of cell types without causing adverse effects. These safe-harbor loci are thus ideal sites for inserting exogenous nucleic acid sequences. Our scientists developed a method for inserting sequences in safe-harbor loci using mRNA encoding gene-editing proteins. Insertion of Sequences into […]
mRNA Delivery to Skin
In addition to being the largest and most accessible organ of the body, the skin contains large numbers of highly active cells that have a high capacity for protein synthesis. These characteristics make the skin an ideal platform for expressing therapeutic proteins, both locally, for the treatment of dermatological conditions, and systemically, for the treatment […]
End-Modified Linear DNA Donors
Gene-editing proteins can be used to inactivate, repair, or insert sequences in living cells. Conventional approaches using plasmids or viruses to encode donor DNA sequences for insertion can result in low-efficiency insertion and unwanted mutagenesis when an exogenous nucleic acid fragment is inserted at random locations in the genome. Our scientists developed a technology that […]
Splint and Ribozyme-Independent Circular RNA Synthesis
RNA molecules can be used to express proteins in cells, both ex vivo and in vivo. Conventional approaches using linear RNA molecules can result in exonuclease degradation, which can lead to low protein expression. Our scientists developed a technology for synthesizing circular RNA molecules that does not require splints or ribozymes9. This technology can enable […]