Chemical Biology and Therapeutics Science
Mission

The primary mission of the Chemical Biology and Therapeutics Science (CBTS) Program at the Ó³»­´«Ã½ is to support community engagement in innovative chemical biology and therapeutics research.

X-ray co-crystal structure of B52 compound (cyan) binding and inhibiting Cyclophilin D protein
Credit: Aziz Rangwala
X-ray co-crystal structure of B52 compound (cyan) binding and inhibiting Cyclophilin D protein

About

The Chemical Biology and Therapeutics Science (CBTS) Program at the Ó³»­´«Ã½ brings together a talented and diverse group of researchers that share a deep commitment to advancing biology and medicine through the use of chemistry and related molecular approaches. Recent advances have expanded the scope of chemical biology and therapeutics science to include: 

  • Breakthroughs in the use of macromolecules to study biology and modify our genomes and proteomes in therapeutically relevant ways.
  • Development of technologies that have transformed how small-molecule and macromolecular structure space can be explored to rapidly identify molecules with remarkable biological properties.
  • Innovative use of chemistry to provide an unprecedented view of previously unseen processes in living systems.
  • Integration of data from multiple technologies and knowledge sources to better understand the roles of small molecules in living systems. 
     

Chemical Biology and Therapeutics Science

Areas of Focus

Recent publications

Other Chemical Biology and Therapeutics Science Program publications
In vivo base editing extends lifespan of a humanized mouse model of prion disease.
Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference.
Post-translational modification-centric base editor screens to assess phosphorylation site functionality in high throughput.
Twin prime editing mediated exon skipping/reinsertion for restored collagen VII expression in recessive dystrophic epidermolysis bullosa.
Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.
Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing.
Uncoupling histone modification crosstalk by engineering lysine demethylase LSD1.
Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells.
Introducing a G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.
EvoAI enables extreme compression and reconstruction of the protein sequence space.

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