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Base editing is unquestionably one of the most exciting recent technology innovations in the field of genome editing. The development of these base editors in the lab of David Liu select Broad Institute/HHMI) enables the substitution of specific bases in DNA select C to T and A to G) without cleaving both strands of the DNA, potentially offering more precision than traditional CRISPR methods. Our speaker for this webinar, Dr. Nicole Gaudelli, led the development of the original A base editor in the Liu lab in 2017 and is currently is spearheading the application of base editing across a range of medical conditions at Beam Therapeutics.

Investigators around the world have seized on these new tools and are pursuing myriad applications from basic research to therapeutics to agricultural biotechnology. Using directed evolution, researchers have also developed a new generation of editors with hundreds of times more activity than the original constructs. Recent results have demonstrated the exciting potential of base editors in animal models for diseases including heart disease and progeria.

During this presentation, which is part of TriLink BioTechnologies gene editing and cell therapy webinar series, Dr. Gaudelli will discuss how her group has transformed off-the-shelf base editors from a technology developed in academia to a promising therapeutic tool through engineering, screening, and directed evolution. Among the topics she will present are:

  • A brief history of the ABCs of base editing
  • Advances in base editing precision, activity, and safety
  • Technical challenges and research priorities
  • Therapeutically relevant application of base editing technologies
  • T cell engineering with adenine base editors, as well as gamma-globin upregulation

A live Q&A session followed the presentation, offering you a chance to pose questions to our expert panelists.

Produced with support from:

Nicole M. Gaudelli, PhD
Associate Director and
Head of Gene Editing Technologies
Beam Therapeutics