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ASGCT 2021 | HSC gene editing: current approaches and remaining challenges

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, provides an overview of the current landscape for hematopoietic stem cell (HSC) gene editing and describes key gene editing approaches used including the non-homologous end joining (NHEJ) and homology-directed repair (HDR) pathways. Key lines of ongoing research to advance HSC gene editing approaches include the improvement of HDR efficiency in HSCs, as well as the development of new tools, such as CRISPR-associated transposases, to circumvent toxicity associated with double-strand breaks. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.