A decade after its initial development, CRISPR genome editing is showing great promise for the treatment of previously intractable diseases, with exciting results reported in early clinical trials. However, challenges in creating precise edits (as opposed to imprecise insertions and deletions) in vivo, and in delivering the editing machinery safely and specifically to target tissues, currently limit therapeutic applications.
In this inaugural Keynote webinar hosted by GEN Biotechnology , Professor Erik Sontheimer presents his team’s advances in developing new CRISPR-Cas effectors, engineered guides, and enhanced templates that improve in vivo implementation across nuclease editing, base editing, and prime editing platforms.