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

Alessia Cavazza; Ayal Hendel; Rasmus O. Bak; Paula Rio; Marc Güell; Duško Lainšček; Virginia Arechavala-Gomeza; Ling Peng; 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; Francisco Martin; Giandomenico Turchiano; Toni Cathomen; Yonglun Luo; Shengdar Q. Tsai; Karim Benabdellah; COST Action CA21113

doi:10.1016/j.omtn.2023.102066

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

Alessia Cavazza^*, Ayal Hendel^*, Rasmus O. Bak^*, Paula Rio, Marc Güell, Duško Lainšček, Virginia Arechavala-Gomeza, Ling Peng, 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 MontoliuMarcello 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, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo^*, Shengdar Q. Tsai^*, Karim Benabdellah^*, COST Action CA21113

^*Corresponding author for this work

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Review › Research › peer-review

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.

Original language	English
Article number	102066
Journal	Molecular Therapy Nucleic Acids
Volume	34
Pages (from-to)	102066
Number of pages	14
ISSN	2162-2531
DOIs	https://doi.org/10.1016/j.omtn.2023.102066 https://doi.org/10.1016/j.omtn.2023.102066
Publication status	Published - 12 Dec 2023

Keywords

base editors
COST
delivery systems
European Cooperation in Science and Technology
GenE-HumDi
genome editing
MT: RNA/DNA Editing
regulatory guidelines

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Cavazza, A., Hendel, A., Bak, R. O., Rio, P., Güell, M., Lainšček, 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., ... COST Action CA21113 (2023). Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi. Molecular Therapy Nucleic Acids, 34, 102066. Article 102066. https://doi.org/10.1016/j.omtn.2023.102066, https://doi.org/10.1016/j.omtn.2023.102066

@article{a0d30758631c42b5b313aa77d477e4c2,

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

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.",

keywords = "base editors, COST, delivery systems, European Cooperation in Science and Technology, GenE-HumDi, genome editing, MT: RNA/DNA Editing, regulatory guidelines",

author = "Alessia Cavazza and Ayal Hendel and Bak, {Rasmus O.} and Paula Rio and Marc G{\"u}ell and Du{\v s}ko Lain{\v s}{\v c}ek and Virginia Arechavala-Gomeza and Ling Peng and Hapil, {Fatma Zehra} and Joshua Harvey and Ortega, {Francisco G.} and Coral Gonzalez-Martinez and Lederer, {Carsten W.} and Kasper Mikkelsen and Giedrius Gasiunas and Nechama Kalter and Gon{\c c}alves, {Manuel A.F.V.} and Julie Petersen and Alejandro Garanto and Lluis Montoliu and Marcello Maresca and Seemann, {Stefan E.} and Jan Gorodkin and Loubna Mazini and Rosario Sanchez and Rodriguez-Madoz, {Juan R.} and Noelia Maldonado-P{\'e}rez and Torella Laura and Michael Schmueck-Henneresse and Cristina Maccalli and Julian Gr{\"u}newald and Gloria Carmona and Neli Kachamakova-Trojanowska and Annarita Miccio and Francisco Martin and Giandomenico Turchiano and Toni Cathomen and Yonglun Luo and Tsai, {Shengdar Q.} and Karim Benabdellah and {COST Action CA21113}",

year = "2023",

month = dec,

day = "12",

doi = "10.1016/j.omtn.2023.102066",

language = "English",

volume = "34",

pages = "102066",

journal = "Molecular Therapy Nucleic Acids",

issn = "2162-2531",

publisher = "Nature Publishing Group",

}

Cavazza, A, Hendel, A, Bak, RO, Rio, P, Güell, M, Lainšček, D, Arechavala-Gomeza, V, Peng, L, Hapil, FZ, Harvey, J, Ortega, FG, Gonzalez-Martinez, C, Lederer, CW, Mikkelsen, K, Gasiunas, G, Kalter, N, Gonçalves, MAFV, Petersen, J, Garanto, A, Montoliu, L, Maresca, M, Seemann, SE, Gorodkin, J, Mazini, L, Sanchez, R, Rodriguez-Madoz, JR, Maldonado-Pérez, N, Laura, T, Schmueck-Henneresse, M, Maccalli, C, Grünewald, J, Carmona, G, Kachamakova-Trojanowska, N, Miccio, A, Martin, F, Turchiano, G, Cathomen, T, Luo, Y, Tsai, SQ, Benabdellah, K & COST Action CA21113 2023, 'Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi', Molecular Therapy Nucleic Acids, vol. 34, 102066, pp. 102066. https://doi.org/10.1016/j.omtn.2023.102066, https://doi.org/10.1016/j.omtn.2023.102066

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi. / Cavazza, Alessia; Hendel, Ayal; Bak, Rasmus O. et al.
In: Molecular Therapy Nucleic Acids, Vol. 34, 102066, 12.12.2023, p. 102066.

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Review › Research › peer-review

TY - JOUR

T1 - Progress and harmonization of gene editing to treat human diseases

T2 - Proceeding of COST Action CA21113 GenE-HumDi

AU - Cavazza, Alessia

AU - Hendel, Ayal

AU - Bak, Rasmus O.

AU - Rio, Paula

AU - Güell, Marc

AU - Lainšček, Duško

AU - Arechavala-Gomeza, Virginia

AU - Peng, Ling

AU - Hapil, Fatma Zehra

AU - Harvey, Joshua

AU - Ortega, Francisco G.

AU - Gonzalez-Martinez, Coral

AU - Lederer, Carsten W.

AU - Mikkelsen, Kasper

AU - Gasiunas, Giedrius

AU - Kalter, Nechama

AU - Gonçalves, Manuel A.F.V.

AU - Petersen, Julie

AU - Garanto, Alejandro

AU - Montoliu, Lluis

AU - Maresca, Marcello

AU - Seemann, Stefan E.

AU - Gorodkin, Jan

AU - Mazini, Loubna

AU - Sanchez, Rosario

AU - Rodriguez-Madoz, Juan R.

AU - Maldonado-Pérez, Noelia

AU - Laura, Torella

AU - Schmueck-Henneresse, Michael

AU - Maccalli, Cristina

AU - Grünewald, Julian

AU - Carmona, Gloria

AU - Kachamakova-Trojanowska, Neli

AU - Miccio, Annarita

AU - Martin, Francisco

AU - Turchiano, Giandomenico

AU - Cathomen, Toni

AU - Luo, Yonglun

AU - Tsai, Shengdar Q.

AU - Benabdellah, Karim

AU - COST Action CA21113

PY - 2023/12/12

Y1 - 2023/12/12

N2 - 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.

AB - 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.

KW - base editors

KW - COST

KW - delivery systems

KW - European Cooperation in Science and Technology

KW - GenE-HumDi

KW - genome editing

KW - MT: RNA/DNA Editing

KW - regulatory guidelines

U2 - 10.1016/j.omtn.2023.102066

DO - 10.1016/j.omtn.2023.102066

M3 - Review

C2 - 38034032

AN - SCOPUS:85176456320

SN - 2162-2531

VL - 34

SP - 102066

JO - Molecular Therapy Nucleic Acids

JF - Molecular Therapy Nucleic Acids

M1 - 102066

ER -

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

Abstract

Keywords

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