• Axel Schambach, Christian J Buchholz, Raul Torres-Ruiz, Klaus Cichutek, Michael Morgan, Ivana Trapani, and Hildegard Büning.
• Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA; REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany; German Center for Infection Research, partner site Hannover-Braunschweig, Germany.
• Lancet. 2024 Feb 10; 403 (10426): 568582568-582.

AbstractGene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of diseases that will become treatable. To frame these impressive advances in the context of modern medicine, we incorporate examples from human clinical trials into our discussion on how genome editing will complement currently available strategies in gene therapy, which still mainly rely on gene addition strategies. Furthermore, safety considerations and ethical implications, including the issue of accessibility, are addressed as these crucial parameters will define the impact that gene therapy in general and genome editing in particular will have on how we treat patients in the near future.Copyright © 2024 Elsevier Ltd. All rights reserved.

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