Scientific publications
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 3, Paula Rio 4 5, Marc Güell 6 7, Duško Lainšček 8, Virginia Arechavala-Gomeza 9 10, Ling Peng 11, Fatma Zehra Hapil 12, Joshua Harvey 13, Francisco G Ortega 14 15, Coral Gonzalez-Martinez 14 15, Carsten W Lederer 16, Kasper Mikkelsen 3, Giedrius Gasiunas 17, Nechama Kalter 2, Manuel A F V Gonçalves 18, Julie Petersen 19, Alejandro Garanto 20, Lluis Montoliu 21, Marcello Maresca 22, Stefan E Seemann 23, Jan Gorodkin 23, Loubna Mazini 24, Rosario Sanchez 25 26 27, Juan R Rodriguez-Madoz 28 29 30, Noelia Maldonado-Pérez 19, Torella Laura 31, Michael Schmueck-Henneresse 32, Cristina Maccalli 33, Julian Grünewald 34 35, Gloria Carmona 36, Neli Kachamakova-Trojanowska 37, Annarita Miccio 38, Francisco Martin 39, Giandomenico Turchiano 1, Toni Cathomen 40 41, Yonglun Luo 3 19, Shengdar Q Tsai 42, Karim Benabdellah 43; COST Action CA21113
Abstract
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.
CITA DEL ARTÍCULO Mol Ther Nucleic Acids. 2023 Oct 29:34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12.