Programming gene circuits with genome and transcriptome engineering to combat disease

Seminar | January 22 | 10-11 a.m. | 290 Hearst Memorial Mining Building

 Patrick Hsu, Salk Institute for Biological Studies

 Bioengineering (BioE)

Rapid advances in genome profiling and analysis have illuminated many genomic changes related to human disease. The ability to interrogate the functional roles of such variants in pathogenesis and their potential as therapeutic targets is critical for the development of new medicines. CRISPR tools that systematically reverse-engineer cellular processes through rapid and precise perturbations enable causal links between genetic changes and fundamental disease mechanisms. I will discuss our efforts to 1) adapt the CRISPR-Cas9 system for precision genome editing, 2) mine the biological diversity as a rich resource of uncharacterized enzyme systems, and 3) discover novel CRISPR-Cas systems to enable a broad suite of transcriptome engineering tools for RNA manipulation. Throughout, I will highlight the promise of molecular technology development to enable new approaches for cell design and control, paving the way for the next generation of gene and cell therapies

 CA,, 5106663362