Dr. Zimmerman's lab has developed a series of bioactive compounds for the treatment of myotonic dystrophy. The compounds disrupt the MBNL-(CUG) foci in vivo and partially rescue the missplicing of cTNT and IR mRNAs. These novel compounds, utilizing bisamidinium-based inhibitors, show a 1000-fold greater inhibition potency than a previous compound and are able to degrade the causative agent of the disease.
Dr. John Rogers from the University of Illinois at Urbana-Champaign has developed bioresorbable silicon electronics that can be used for real-time sensing of neural electrical activity. This invention could prevent follow-up neural surgeries, and has potentials for long-term monitoring of patients.
Dr. Andrew Smith from the University of Illinois has developed new quantum dots with a multidentate polymer coating that minimizes size while maintaining stability and improving efficiency of conjugation. Quantum dots are promising agents for cellular and molecular imaging, but their bulky organic coatings have limited their use in cells. Dr. Smith's quantum dots are small, stable, and can be conjugated to targeting molecules and purified easily.
Dr. Hergenrother from the University of IL has developed a novel antibiotic that is effective against certain antibiotic-resistant gram-negative bacteria. His powerful predictive algorithm determines accumulation of molecules in Gram-negative bacteria and enables conversion of known Gram-positive only antibiotics into novel compounds with Gram-negative potency.
Dr. Zimmerman from the University of Illinois at Urbana-Champaign has developed an oligomeric ligand that can have potential applications in the treatment of DM1. This Ligand on account of its multivalent nature, is an improvement over the previous small molecule (TF12039) designed by the inventor. The oligomeric ligand has been shown to rescue mis-splicing activity in DM1 cell models. Climbing defects in adult DM1 drosophila flies was also show to have been rescued on treatment with this oligomeric ligand.
Dr. Perez-Pinera has developed a new way to edit genes using CRISPR technology that promises to be safer and more efficient that existing technologies. His method uses single base editing, to avoid double-stranded breaks in DNA and induce a mutation that causes exon skipping to occur which can silence, knockdown or ameliorate the effects of downstream gene products. Avoiding double stranded breaks eliminates the possibility of malignant mutations which can result from improper non-homologous end joining. He also demonstrates a novel delivery method for the CRISPR-cas9 system using a split base editor inserted into an adeno-associated virus to realize greater, more efficacious delivery into the cell. This method can be used to treat diseases such as Duchenne’s Muscular Dystrophy with more permanence than current treatments as well as Huntington’s and cardiovascular diseases.