*** NOTE: Time & Date is THURSDAY, FEB. 9th @ 2:00 PM ***
Genetic disorders are a final frontier in human disease and medicine. CRISPR-Cas systems represent the most promising therapeutic candidates in development for genomic medicines that can correct genetic mutations. Despite their promise, they are controversial and challenging to develop due to the danger of unanticipated and irreversible changes to a patient’s genome. Their controllability, safety, and – as rather large biologics – deliverability, stand as remaining barriers to their eventual success. This seminar will discuss progress toward full chemical modification of CRISPR-Cas9 and CRISPR-Cas12a guide RNAs for better tunability and deliverability as well as development of novel inhibitors to improve safety.