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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

Autores: Cavazza, A. (Autor de correspondencia); Hendel, A. (Autor de correspondencia); Bak, R. O. (Autor de correspondencia); Rio, P.; Güell, M.; Lainscek, D.; Arechavala-Gomeza, V.; Peng, L.; Hapil, F. Z.; Harvey, J.; Ortega, F. G.; Gonzalez-Martinez, C.; Lederer, C. W.; Mikkelsen, K.; Gasiunas, G.; Kalter, N.; Gonçalves, M. A. F. V.; Petersen, J.; Garanto, A.; Montoliu, L.; Maresca, M.; Seemann, S. E.; Gorodkin, J.; Mazini, L.; Sanchez, R.; Rodríguez Madoz, Juan Roberto; Maldonado-Pérez, N.; Torella, Laura; Schmueck-Henneresse, M.; Maccalli, C.; Grünewald, J.; Carmona, G.; Kachamakova-Trojanowska, N.; Miccio, A.; Martin, F.; Turchiano, G.; Cathomen, T.; Luo, Y. L. (Autor de correspondencia); Tsai, S. Q. (Autor de correspondencia); Benabdellah, K. (Autor de correspondencia)
Título de la revista: MOLECULAR THERAPY - NUCLEIC ACIDS
ISSN: 2162-2531
Volumen: 34
Páginas: 102066
Fecha de publicación: 2023
Resumen:
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 com-panies, 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 reg-ulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scien-tific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome edit-ing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Gran-ada, 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.
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