From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications

Ortiz-Bueno, María; Zinghirino, Federica; Serra, Pilar Puig; Paschoudi, Kyriaki; Montoliu, Lluis; Atilla, Erden; Luo, Yonglun; Cavazza, Alessia; Lederer, Carsten W.; Benabdellah, Karim

doi:10.3390/ijms27031484

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From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications

by

María Ortiz-Bueno

¹

,

Federica Zinghirino

²

,

Pilar Puig Serra

³

,

Kyriaki Paschoudi

^4,5,

Lluis Montoliu

^6,7,

Erden Atilla

^1,8

,

Yonglun Luo

^9,10

,

Alessia Cavazza

^11,12,

Carsten W. Lederer

^13,*

and

Karim Benabdellah

^1,*

¹

GENYO, Centre for Genomics and Oncological Research: Pfizer, University of Granada, 18016 Granada, Spain

²

Department of Biochemical Engineering, University College London (UCL), London WC1E 6BT, UK

³

Molecular Cytogenetics and Genome Editing Unit, Human Cancer Genetics Program, Spanish National Cancer Research Center (CNIO), 28029 Madrid, Spain

⁴

Department of Genetics, Development and Molecular Biology, School of Biology, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece

⁵

Gene and Cell Therapy Center, Hematology Clinic, George Papanikolaou Hospital, 57010 Thessaloniki, Greece

⁶

Department of Molecular and Cellular Biology, National Centre for Biotechnology (CNB-CSIC), 28049 Madrid, Spain

⁷

Rare Diseases Networking Biomedical Research Centre (CIBERER-ISCIII), 28029 Madrid, Spain

⁸

Transplantation and Cellular Therapy, Department of Medicine, University of Miami Miller School of Medicine, Miami, FL 33136, USA

⁹

Department of Biomedicine, Aarhus University, 8000 Aarhus, Denmark

¹⁰

Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus, Denmark

¹¹

Molecular and Cellular Immunology Section, Department of Infection, Immunity & Inflammation, Great Ormond Street Institute of Child Health, University College London, London WC1N 1DZ, UK

¹²

Department of Medical and Surgical Sciences for Children and Adults, School of Medicine, University of Modena and Reggio Emilia, 41124 Modena, Italy

¹³

Department of Blood Disorder Genetics & Thalassemia, The Cyprus Institute of Neurology & Genetics, Nicosia 2371, Cyprus

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^*

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Int. J. Mol. Sci. 2026, 27(3), 1484; https://doi.org/10.3390/ijms27031484

Submission received: 30 October 2025 / Revised: 20 January 2026 / Accepted: 21 January 2026 / Published: 2 February 2026

(This article belongs to the Special Issue Application of Genetic Engineering in Treatments for Human Diseases)

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Abstract

Genome editing (GE) has transformed medicine by allowing precise changes to DNA, offering potential treatments for a range of inherited and acquired disorders. Several technologies support these advances, including zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)-based systems, of which the latter has emerged as the most accessible, versatile, and popular. While GE holds great promise, its clinical use requires careful attention to safety, ethics and regulatory standards. Inadvertent on- and off-target DNA alterations and unintended modification of non-target cells pose major technical challenges, while bioethical considerations and the need for harmonized safety standards create regulatory challenges. The Food and Drug Administration (FDA) and European Medicines Agency (EMA), as regulatory agencies for key advanced therapy markets, provide detailed guidance on these aspects, emphasizing rigorous preclinical testing, patient monitoring, ethical consent, and compliance with legal frameworks. This concise review summarizes what is currently published in the scientific literature and recommended by regulatory agencies, providing an overview of the responsible clinical application of GE, with emphasis on patient safety, adherence to regulatory guidance, and ethical practice.

Keywords: genome editing; CRISPR; best practices; clinical applications; off-target effects

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MDPI and ACS Style

Ortiz-Bueno, M.; Zinghirino, F.; Serra, P.P.; Paschoudi, K.; Montoliu, L.; Atilla, E.; Luo, Y.; Cavazza, A.; Lederer, C.W.; Benabdellah, K. From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications. Int. J. Mol. Sci. 2026, 27, 1484. https://doi.org/10.3390/ijms27031484

AMA Style

Ortiz-Bueno M, Zinghirino F, Serra PP, Paschoudi K, Montoliu L, Atilla E, Luo Y, Cavazza A, Lederer CW, Benabdellah K. From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications. International Journal of Molecular Sciences. 2026; 27(3):1484. https://doi.org/10.3390/ijms27031484

Chicago/Turabian Style

Ortiz-Bueno, María, Federica Zinghirino, Pilar Puig Serra, Kyriaki Paschoudi, Lluis Montoliu, Erden Atilla, Yonglun Luo, Alessia Cavazza, Carsten W. Lederer, and Karim Benabdellah. 2026. "From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications" International Journal of Molecular Sciences 27, no. 3: 1484. https://doi.org/10.3390/ijms27031484

APA Style

Ortiz-Bueno, M., Zinghirino, F., Serra, P. P., Paschoudi, K., Montoliu, L., Atilla, E., Luo, Y., Cavazza, A., Lederer, C. W., & Benabdellah, K. (2026). From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications. International Journal of Molecular Sciences, 27(3), 1484. https://doi.org/10.3390/ijms27031484

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