ISTH 2023 | Autologous CRISPR/Cas9 correction of a missense variant in inherited FVII deficiency
Giacomo Roman, MSc, Oslo University Hospital, Oslo, Norway, discusses the recent generation of autologous induced pluripotent stem cell (iPSC) lines from two patients’ primary cells which allowed CRISPR/Cas9 correction of a missense mutation in the factor VII (FVII) gene. The iPSC lines were successfully generated from both patients using a novel and robust method to allow generation of autologous cell lines from a single cell, eliminating the possibility of mixed cell populations, which are often a limitation for iPSC and gene therapies. Dr Roman explains how these results can open the doors for regenerative medicines in the treatment of many bleeding disorders. This interview took place at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Montréal, Canada.
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