Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
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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.
| Originalsprog | Engelsk |
|---|---|
| Artikelnummer | 102066 |
| Tidsskrift | Molecular Therapy Nucleic Acids |
| Vol/bind | 34 |
| Antal sider | 14 |
| ISSN | 2162-2531 |
| DOI | |
| Status | Udgivet - 2023 |
Bibliografisk note
Funding Information:
This publication is based upon work from COST Action Gene Editing for the treatment of Human Diseases, CA21113 (https://www.genehumdi.eu) supported by COST (European Cooperation of Science and Technology). The study was also supported by the Consejería de Universidad, Investigación e Innovación under Plan Andaluz de Investigación, Desarrollo e Innovación (PAIDI 2020) (ProyExcel_00875) de la junta de Andalucía, and by Consejería de Salud y familia de la Junta de Andalucia/FEDER (PECART-0027-2020), K.B. held Nicolas Monardes contract from Consejería de Salud y Consumo de la Junta de Andalucía. A.C.: Conceptualization, data curation, Network administration, investigation, visualization, writing—original draft, writing—review and editing. A.H. R.O.B. P.R. M.G. D.L. V.A. L.P. F.Z.H. J.H. F.G.O. C.G.M. C.L. K.M. G.G. N.K. M.A.F.V.G.; A.G. L.M. M.M, J.G. M.L. R.S. J.R.R.M. N.M.P. L.T. M.S.H. C.M. J.G. G.C. A.M. F.M. G.T. T.C. Y.L. T.Q.S.: validation, review editing. K.B.: Conceptualization, funding acquisition, investigation, Network administration, resources, supervision, visualization, writing—review. P.R. has licensed the PGK:FANCAWpre∗ LV medicinal product and receives funding and equity from Rocket Pharmaceuticals, Inc. patents and royalties, research & consulting funding. D.L. is an inventor on a patent National Institute of Chemistry filed (WO/2021/032759 patent application, European patent application EP 3783104, China patent application CN 114269930 with National Phase entry EP2020756868). R.O.B. holds patents related to CRISPR-Cas genome editing and has equity in Graphite Bio and is consultant for UNIKUM Tx. G.G. holds patents related to CRISPR-Cas genome editing, is an employee of CasZyme, and has equity in CasZyme. S.Q.T. is a co-inventor on patents for GUIDE-seq, CHANGE-seq, and other genome editing technologies and a member of the scientific advisory boards of Prime Medicine and Ensoma. T.C. is a co-inventor on patents for CAST-seq, Abnoba-Seq, and other genome editing technologies, and a member of the scientific advisory boards of Cimeo Therapeutics, Excision BioTherapeutics, and GenCC. A.C. and G.T. are inventors on a patent for MEGA (WO/2023/079285), G.T. is also co-inventor on a patent for CAST-seq.
Funding Information:
This publication is based upon work from COST Action Gene Editing for the treatment of Human Diseases, CA21113 ( https://www.genehumdi.eu ) supported by COST (European Cooperation of Science and Technology). The study was also supported by the Consejería de Universidad, Investigación e Innovación under Plan Andaluz de Investigación, Desarrollo e Innovación ( PAIDI 2020 ) ( ProyExcel_00875 ) de la junta de Andalucía , and by Consejería de Salud y familia de la Junta de Andalucia /FEDER ( PECART-0027-2020 ), K.B. held Nicolas Monardes contract from Consejería de Salud y Consumo de la Junta de Andalucía.
Publisher Copyright:
© 2023 The Author(s)
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