Progress and Harmonization of Gene Editing to Treat Human Diseases (Proceeding of COST Action CA21113 GenE-HumDi)

Alessia Cavazza (1) , Ayal Hendel (2) , Rasmus O Bak , Paula Rio , Marc Güell , Duško Lainšček , Virginia Arechavala-Gomeza (3) , Ling Peng (4) , Fatma Zehra Hapil , Joshua Harvey , Francisco G Ortega , Coral Gonzalez-Martinez , Carsten W Lederer , Kasper Mikkelsen , Giedrius Gasiunas , Nechama Kalter , Manuel a F V Gonçalves , Julie Petersen , Alejandro Garanto , Lluis Montoliu , Marcello Maresca , Stefan E Seemann , Jan Gorodkin , Loubna Mazini , Rosario Sanchez , Juan R Rodriguez-Madoz , Noelia Maldonado-Pérez , Torella Laura , Michael Schmueck-Henneresse , Cristina Maccalli , Julian Grünewald , Gloria Carmona , Neli Kachamakova-Trojanowska , Annarita Miccio (5) , Francisco Martin , Giandomenico Turchiano , Toni Cathomen , Yonglun Luo , Shengdar Q Tsai , Karim Benabdellah

1 UCL - University College of London [London]
2 Bar-Ilan University [Israël]
3 Biocruces Bizkaia Health Research Institute [Baracaldo]
4 CINaM - Centre Interdisciplinaire de Nanoscience de Marseille
5 Imagine - U1163 - Imagine - Institut des maladies génétiques (IHU)

The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the "Genome Editing to treat Human Diseases" (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi's primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.

Mots clés: Molecular Therapy: Nucleic Acid

Domaines: Génomique, Transcriptomique et Protéomique [q-bio.GN]; Chimie thérapeutique

Alessia Cavazza, Ayal Hendel, Rasmus O Bak, Paula Rio, Marc Güell, et al.. Progress and Harmonization of Gene Editing to Treat Human Diseases (Proceeding of COST Action CA21113 GenE-HumDi). Molecular Therapy - Nucleic Acids, 2023, ⟨10.1016/j.omtn.2023.102066⟩. ⟨hal-04283508⟩