RNA and DNA based therapeutics

The recent successes of RNA- and DNA-based therapeutics have awakened an interest in exploring the use of oligonucleotide drugs (ONDs) to manipulate human biology. Because many applications of ONDs require placement of the drug specifically in a desired pathologic cell type, progress on these applications has been slowed by an inability to target ODN delivery. We have recently exploited our large toolbox of cell-targeting ligands to concentrate attached ONDs specifically in diseased cells in order to develop novel therapies for cancers and age-related macular degeneration.  The graph below demonstrates our ability to suppress tumor growth with a microRNA termed miR34a (in collaboration with Dr. Andrea Kasinski). The image shows our ability to target a fluorescent siRNA specifically to the retinal pigment epithelium (RPE) of the eye in a live mouse as a method to suppress AMD.