Progress and Harmonization of Gene Editing to Treat Human Diseases (Proceeding of COST Action CA21113 GenE-HumDi) - Archive ouverte HAL Access content directly
Journal Articles Molecular Therapy - Nucleic Acids Year : 2023

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

Rasmus O Bak
  • Function : Author
Paula Rio
  • Function : Author
Marc Güell
  • Function : Author
Duško Lainšček
  • Function : Author
Fatma Zehra Hapil
  • Function : Author
Joshua Harvey
  • Function : Author
Francisco G Ortega
  • Function : Author
Coral Gonzalez-Martinez
  • Function : Author
Carsten W Lederer
  • Function : Author
Kasper Mikkelsen
  • Function : Author
Giedrius Gasiunas
  • Function : Author
Nechama Kalter
  • Function : Author
Manuel a F V Gonçalves
  • Function : Author
Julie Petersen
  • Function : Author
Alejandro Garanto
  • Function : Author
Lluis Montoliu
  • Function : Author
Marcello Maresca
  • Function : Author
Stefan E Seemann
  • Function : Author
Jan Gorodkin
  • Function : Author
Loubna Mazini
  • Function : Author
Rosario Sanchez
  • Function : Author
Juan R Rodriguez-Madoz
  • Function : Author
Noelia Maldonado-Pérez
  • Function : Author
Torella Laura
  • Function : Author
Michael Schmueck-Henneresse
  • Function : Author
Cristina Maccalli
  • Function : Author
Julian Grünewald
  • Function : Author
Gloria Carmona
  • Function : Author
Neli Kachamakova-Trojanowska
  • Function : Author
Francisco Martin
  • Function : Author
Giandomenico Turchiano
  • Function : Author
Toni Cathomen
  • Function : Author
Yonglun Luo
  • Function : Author
Shengdar Q Tsai
  • Function : Author
Karim Benabdellah
  • Function : Author

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.
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Dates and versions

hal-04283508 , version 1 (13-11-2023)

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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⟩
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