At UT Southwestern, the Siegwart Research Group uses a materials chemistry approach to enable targeted nanoparticle delivery of genomic medicines.
Our efforts led to an understanding of the essential physical and chemical properties of synthetic carriers required for therapeutic delivery of siRNA, miRNA, tRNA, mRNA, sgRNA, pDNA, proteins, and gene editors. Our lab has been at the forefront in the design of synthetic carriers for gene editing and has applied these technologies for correction of genetic diseases and treatment of cancer. We reported the first non-viral system for in vivo CRISPR/Cas gene editing. Recently, we developed Selective ORgan Targeting (SORT) lipid nanoparticles (LNPs), which was the first strategy for predictable tissue specific mRNA delivery and gene editing. We ultimately aspire to utilize chemistry and engineering to make a beneficial impact on human health.