RNA-guided CRISPR-Cas nucleases are a transformative genome-editing tool due to the simplicity with which they can be programmed to target new sites. Genome editing has the potential to fulfill medicine’s long-held aspiration to directly address the genetic causes of human diseases. An important step in translating these powerful technologies into safe, effective genomic medicines is understanding their activity and their recognition of on- and off-target sites throughout the genome. In recent years, a number of cellular and biochemical approaches have been developed to characterize the genome-wide activity of CRISPR-Cas genome editors. However, the basic principles governing their specificity remain undefined because current approaches cannot analyze the large numbers of targets required to understand these principles. In this webinar, we review considerations for evaluating the safety of genome editors for therapeutics as well as existing methods for defining the specificity of genome editors, and describe a novel method called Circularization for High-throughput Analysis of Nuclease Genome-wide Effects by sequencing, or CHANGE-seq. Using CHANGE-seq, we can define both genetic and epigenetic factors that affect genome-wide activity in therapeutically relevant human primary T cells.
During the webinar, viewers will:
- Learn how advances in genome-editing technology could potentially address genetic disorders
- Increase their understanding of CRISPR-Cas genome editing activity and safety
- Hear about CHANGE-seq, a novel high-throughput method to define the genome-wide activity of editors, and its application for understanding genetic and epigenetic factors impacting CRISPR-Cas9 activity
- Have the opportunity to ask questions during the live event.
This Webinar will last for approximately 60 minutes.