The Sullenger Laboratory strives to develop novel, safe and effective nucleic acid therapeutics. The lab currently focuses on two areas: RNA/DNA repair via targeted trans-splicing and the development of RNA ligands to protein targets to block/alter their function.

 

 

1. RNA repair: Using spliceosome-mediated or group I ribozyme-directed approaches

2. DNA repair: Using the group II mobile intron

 

* 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

 

 

 

 

 

* In-vitro selection & combinatorial chemistry

* Cell cycle modulator

* Immune response modulator

* Anticoagulation

* For targeted delivery of siRNAs

The SELEX Process