The Cardiovascular Biology Laboratory, under the direction of Bruce Sullenger, is focused on multidisciplinary translational research approaches to the study of blood coagulation, inflammation, and atherogenesis at the molecular level. Novel anti-coagulation approaches developed within the program are presently undergoing pre-clinical and clinical evaluation. Ongoing studies are aimed at exploring molecular therapeutic approaches in the treatment of cardiovascular disease.
The lab strives to develop novel, safe, and effective nucleic acid therapeutics. The lab currently focuses on two areas:
- RNA and DNA repair via targeted trans-splicing
- The development of RNA ligands to protein targets to block or alter their function
RNA and DNA Repair by Targeted Trans-Splicing
- RNA repair: Using spliceosome-mediated or group I ribozyme-directed approaches
- DNA repair: Using the group II mobile intron
Goals for each approach
- Evaluate and enhance activity in mammalian cell lines, primary cells, and animal models
- Apply to clinically relevant targets such as beta-globin (sickle cell anemia, beta-thalassemia), p53 (cancer), mitochondrial genome (certain muscular/neurologic conditions), CCR5 (HIV)
- Uncover factors influencing trans-splicing and reactions specific to the group II mobile intron
RNA Ligands To Block or Alter Target Protein Functions
- In-vitro selection and combinatorial chemistry
- Cell cycle modulator
- Immune response modulator
- Anticoagulation
- For targeted delivery of siRNAs