Evidence map›Paper›PMID 38034032›Full record

ReviewMolecular therapy. Nucleic acids2023

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 and 31 more

Abstract readReview
In one paragraph

Review in Molecular therapy. Nucleic acids, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed.

  1. Review
  2. Article
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  10. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

41 authors.

Alessia CavazzaInfection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK.
Ayal HendelInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel.
Rasmus O BakDepartment of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.
Paula RioHematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIEMAT/CIBERER), 28040 Madrid, Spain.
Marc GüellDepartment of Medicine and Life Sciences, Universitat Pompeu Fabra, Barcelona, Spain.
Duško LainščekDepartment of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia.
Virginia Arechavala-GomezaNucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute, Barakaldo, Spain.
Ling PengAix Marseille University, CNRS, Centre Interdisciplinaire de Nanoscience de Marseille, UMR 7325, Equipe Labellisée Ligue Contre le Cancer, 13288 Marseille, France.
Fatma Zehra HapilDepartment of Medical Biology and Genetics, Faculty of Medicine, Akdeniz University, Antalya, Turkey.
Joshua HarveyInstitute of Ophthalmology, University College London, London, UK.
Francisco G OrtegaGENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, Avenida de la Ilustración 114, 18016 Granada, Spain.
Coral Gonzalez-MartinezGENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, Avenida de la Ilustración 114, 18016 Granada, Spain.
Carsten W LedererDepartment of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Kasper MikkelsenDepartment of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.
Giedrius GasiunasCasZyme, 10224 Vilnius, Lithuania.
Nechama KalterInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel.
Manuel A F V GonçalvesDepartment of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.
Julie PetersenSteno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark.
Alejandro GarantoDepartment of Pediatrics and Department of Human Genetics, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, the Netherlands.
Lluis MontoliuDepartment of Molecular and Cellular Biology, National Centre for Biotechnology (CNB-CSIC) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain.
Marcello MarescaGenome Engineering, Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
Stefan E SeemannCenter for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark.
Jan GorodkinCenter for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark.
Loubna MaziniLaboratory of Genetic Engineering, Technologic, Medical and Academic Park (TMAP), Marrakech, Morocco.
Rosario SanchezGENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, PTS Granada, Granada, Spain.
Juan R Rodriguez-MadozCancer Center Clinica Universidad de Navarra (CCUN), Pamplona, Spain.
Noelia Maldonado-PérezSteno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark.
Torella LauraDNA & RNA Medicine Division, Center for Applied Medical Research (CIMA) Universidad de Navarra, 31008 Pamplona, Spain.
Michael Schmueck-HenneresseBerlin Institute for Health (BIH) at Charité - Universitätsmedizin Berlin, BIH Center for Regenerative Therapies (BCRT), Charitéplatz 1, 10117 Berlin, Germany.
Cristina MaccalliLaboratory of Immune Biological Therapy, Research Branch, Sidra Medicine, PO Box 26999, Doha, Qatar.
Julian GrünewaldDepartment of Medicine, Cardiology, Angiology, Pneumology, Klinikum rechts der Isar, Technical University of Munich, TUM School of Medicine and Health, TranslaTUM, MIBE, Munich, Germany.
Gloria CarmonaRed Andaluza de diseño y traslación de Terapias Avanzadas-RAdytTA, Fundación Pública Andaluza Progreso y Salud-FPS, Sevilla, España.
Neli Kachamakova-TrojanowskaMalopolska Centre of Biotechnology, Jagiellonian University, 30-387 Krakow, Poland.
Annarita MiccioImagine Institute, Laboratory of Chromatin and Gene Regulation During Development, Université de Paris Cité, INSERM UMR 1163, 75015 Paris, France.
Francisco MartinBioquímica y Biología Molecular III e Immunology Department, Facultad de Medicina, Universidad de Granada and Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), PTS, Av. de la Ilustración 114, 18016 Granada, Spain.
Giandomenico TurchianoInfection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK.
Toni CathomenInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, Freiburg, Germany.
Yonglun LuoDepartment of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Karim BenabdellahDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), PTS, Av. de la Ilustración 114, 18016 Granada, Spain.
COST Action CA21113

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

base editorsCOSTdelivery systemsEuropean Cooperation in Science and TechnologyGenE-HumDigenome editingMT: RNA/DNA Editingregulatory guidelines

Identifiers

PMID38034032
PMCPMC10685310

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.