The breakthrough technology CRISPR/Cas has revolutionized molecular approaches to the treatment of disease and provided opportunities for the development of clinically relevant gene therapies. While a wealth of information surrounding gene knockout in human cells is now in hand, we are only beginning to appreciate the intricacies and genetic outcomes resulting from reactions where the aim is to correct a mutation or precisely tag a specific sequence in the genome.
The most well-known method for gene repair is homology-directed repair (HDR), a heterogeneous series of pathways whose activity lead to precise and imprecise genetic alterations. Some pathway activity can lead to unwanted changes.
In this webinar, Eric Kmiec, founder of the Gene Editing Institute at ChristianaCare, will discuss the global distribution of genetic outcomes as a function of CRISPR-directed gene editing, highlighting in vitro and in vivo systems that enable such visualization.
An appreciation for the heterogeneity of HDR products in a whole cell population is becoming essential as therapeutic applications of gene repair advance toward the clinic.