ASGCT 2021 | HSC gene editing highlights from ASGCT 2021

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, discusses her highlights from the American Society for Cell & Gene Therapy (ASGCT) Annual Meeting 2021. Exciting advances in gene editing technology include the development of CRISPR-associated transposase systems and prime editing, which can overcome toxicities associated with double-strand breaks in hematopoietic stem cells. In vivo genome editing is another promising area of research, despite safety and efficacy challenges that still need to be addressed before this technology can be used in the clinic. Dr Vavassori also highlights ongoing research into the potential of adverse events and toxicity caused by genome editing, including chromothripsis and translocations, as gene therapies approach clinical approval. This interview took place during the ASGCT 24th Annual Meeting 2021.