2026-06-30T01:54:04Zhttps://repisalud.isciii.es/rest/oai/request

oai:repisalud.isciii.es:20.500.12105/261782025-12-18T12:55:54Zcom_20.500.12105_2327com_20.500.12105_2290col_20.500.12105_16991

00925njm 22002777a 4500 dc Schambach, Axel author Buchholz, Christian J author Torres-Ruiz, Raul author Cichutek, Klaus author Morgan, Michael author Trapani, Ivana author Büning, Hildegard author Torres-Ruiz, Raul author 2024-02-10 Gene 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. Lancet . 2024 Feb 10;403(10426):568-582. Lancet 38006899 https://hdl.handle.net/20.500.12105/26178 IN-VIVO T-CELLS GENOMIC-DNA BASE BACTERIAL EFFICACY VECTORS PHASE-1 SYSTEM A new age of precision gene therapy.