GetRadi
The GetRadi project ended 30 August 2026
Publications
Mentani A, Maresca M, Shiriaeva A. Prime Editing: Mechanistic Insights and DNA Repair Modulation. Cells. 2025 Feb 13;14(4):277. doi: 10.3390/cells14040277.PMID: 39996750.
Dacquay LC, Antoniou P, Mentani A, Selfjord N, Mårtensson H, Hsieh PP, Mustfa S, Thom G, Wimberger S, Firth M, Akrap N, Maresca M, Peterka M. Dual inhibition of DNA-PK and Polϴ boosts precision of diverse prime editing systems. Nat Commun. 2025 May 8;16(1):4290. doi: 10.1038/s41467-025-59708-z.PMID: 40341582
Stigzelius V, Cavallo AL, Chandode RK, Nitsch R. Peeling back the layers of immunogenicity in Cas9-based genomic medicine. Mol Ther. 2025 Oct 1;33(10):4714-4730. doi:10.1016/j.ymthe.2025.06.045. Epub 2025 Jul 1.PMID: 40605271
Kokavec J, Turková T, Schuster B, Prochazka J, Spoutil F, Jamrichová K, Holečková M, Chalupský K, Beck IM, Ruberte J, Vale M, Čermák L, Stopka T, Sedlacek R. Rps19 R67∆ mutation creates a model of Diamond-Blackfan anemia and reveals downstream mediators of p53 pathway. Hemasphere. 2026 Jan 19;10(1):e70302. doi: 10.1002/hem3.70302. eCollection 2026 Jan.PMID: 41560850
Noujarède J, Wang Q, Kokkinogenis EP, Luo Y, Cudina I, Willaume S, Mooser C, Nguyen LP, Poulsen MF, Heijmerikx S, Le Phan TH, He X, Andersen JB, Sørensen CS, Brakebusch C. RhoA wild-type allele loss unleashes the cancer driver function of RhoA E40Q. J Clin Invest. 2026 Aug 25:e201975. doi: 10.1172/JCI201975. Online ahead of print.PMID: 42640717
Matilde Vale, Jan Prochazka, Radislav Sedlacek. Towards a Cure for Diamond–Blackfan Anemia: Views on Gene Therapy. Cells, https://doi.org/10.3390/cells13110920
Maryam Taghdiri, Claudio Mussolino. Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets. International Journal of Molecular Sciences, https://doi.org/10.3390/ijms25137333
Xiubin He, Cord Brakebusch. Regulation of Precise DNA Repair by Nuclear Actin Polymerization: A Chance for Improving Gene Therapy? Cells, https://doi.org/10.3390/cells13131093
Aurora Giommetti, Eleni Papanikolaou. Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction. Cells, https://doi.org/10.3390/cells13121039
Wang, Qian; Capelletti, Sabrina; Liu, Jin; Janssen, Josephine; Goncalves, Manuel. Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells. Nucleic Acid Research, https://doi.org/10.1093/nar/gkae057
Sabrina Capelletti, Sofía C. García Soto, Manuel A.F.V. Gonçalves. On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies. Molecular Therapy, https://doi.org/10.1016/j.ymthe.2024.08.016
Daniel Bauersachs, Louise Bomholtz, Sara del Rey Mateos, Ralf Kühn and Pawel Lisowski. Novel human neurodevelopmental and neurodegenerative disease associated with IRF2BPL gene variants—mechanisms and therapeutic avenues. Frontiers in Neuroscience, https://doi.org/10.3389/fnins.2024.1426177
Siang Li, Cord Brakebusch. Reporter Mice for Gene Editing: A Key Tool for Advancing Gene Therapy of Rare Diseases. Cells, https://doi.org/10.3390/cells13171508
A rare disease is a disease that affects less than 1 in 2000 people.
However, since more than 6000 rare diseases are known, approximately 300 million people are suffering from rare diseases worldwide - including 30 million in the EU alone. Most of these rare diseases are caused by a genetic mutation and therefore a genetic therapy based on gene augmentation or, ideally, direct gene correction, is the only possibility to offer a permanent cure. However, only very few gene therapy products have reached the market up to now. While this is proof-of-principle for the potential of gene therapies, it also illustrates that there is an urgent need for the development of many more of them, especially at more affordable prices.
To achieve this, novel ideas are required to solve different technical problems associated with multifaceted nature of gene therapy, but also researchers with an excellent education in all technologies associated with gene therapies including rare disease models. Therefore, the overall goal of our network Gene Therapy of Rare Diseases (GetRadi) is to train 10 PhD students to become exceptional experts in gene therapy of rare diseases. In their research projects, the ESR will aim to overcome major technical challenges that still prevent the widespread use of somatic gene therapy in the clinic. All projects are embedded in a tight and synergistic academic-industrial collaboration to develop marketable products for the application of gene therapy. This GetRadi training will allow young scientists to spearhead future research efforts in gene therapy of rare diseases and thus increase the speed that desperately needed novel gene therapies are developed.
Funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them.
Beneficiaries
University of Copenhagen (UCPH), Denmark
Cord Brakebusch
Professor
BRIC
cord.brakebusch@bric.ku.dk
Phone: +45 3532 5169
Hans H. Wandall
Professor, MD, PhD
Centre for Glycomics, Department of Cellular and Molecular Medicine
hhw@sund.ku.dk
Phone: + 45 2721 0936
AstraZeneca AB (AZ), Sweden
Marcello Maresca
Senior Director
AstraZeneca, Pepparedsleden 1, 431 50 Mölndal, Göteborg, Sweden
Marcello.Maresca@astrazeneca.com
Phone: +46 7063 15840
Freiburg University Medical Center (UKLFR), Germany
Claudio Mussolino
Group Leader “Genome and Epigenome Engineering”
Medical Center - University of Freiburg, Institute for Transfusion Medicine and Gene Therapy & Center for Chronic Immunodeficiency at Center for Translational Cell Research (ZTZ). Breisacherstr. 115, 79106 Freiburg, Germany
claudio.mussolino@uniklinik-freiburg.de
Phone: +49 761 270-77738
Leiden University Medical Center (LUMC), The Netherlands
Manuel A.F.V. Gonçalves
Associate professor
Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20 2333 ZC Leiden, The Netherlands.
m.f.v.goncalves@lumc.nl
Phone: +31 71 52 69238
Max Delbrück Center (MDC), Germany
Ralf Kühn
Staff scientist
Max Delbrück Center for Molecular Medicine, Robert-Rössle-Str.10, 13125 Berlin, Germany
ralf.kuehn@mdc-berlin.de
Phone: 030-9406-2832
Institute for Molecular Genetics (IMG), Czech Republic
Radislav Sedlacek
Director of the Czech Centre for Phenogenomics, head of the laboratory of transgenic models of diseases
Institute of Molecular Genetics of the ASCR, v. v. i., Prumyslova 595, 252 50 Vestec
radislav.sedlacek@img.cas.cz
Phone: (+420) 325 873 243-2
Miltenyi Biotec BV & Co (MB), Germany
Eleni Papanikolaou
Strategic Lead, Hemopoietic Stem Cells, Department of Research and Development, Miltenyi Biotec
Friedrich-Ebert-Straße 68, 51429 Bergisch Gladbach, Germany
elenip@miltenyi.com
Phone +49 2204 8306-0
Associated partners
Synvolux Therapeutics BV, The Netherlands
Dr. Gerben C.M. Zondag
CEO
J.H. Oortweg 19, 2333 CH, Leiden, The Netherlands
g.zondag@synvolux.com
Phone: +31 640209098
Amniotics AB, Sweden
Jan Talts
COO
Amniotics AB, Scheelevägen 2, Building 406, SE-223 81 Lund, Sweden
jt@amniotics.com
Phone: +46 72-327 8530
EuroGCT / The University of Edinburgh, United Kingdom
Amanda Waite
Project Manager – Information and Networks - EuroGCT
The European Consortium for Communicating Gene and Cell Therapy Information.
Centre for Regenerative Medicine, Institute for Regeneration and Repair, The University of Edinburgh, Edinburgh BioQuarter, 5 Little France Drive, Edinburgh EH16 4UU
euroenq@ed.ac.uk
Ten fellows were recruited within the GetRadi network to receive exceptional local and network-wide training.
Meet the future experts on gene therapy of rare diseases:
DC1 - Ana Matilde Augusto Vale
Name: Matilde Vale
Nationality: Portuguese
Academic Background: B.Sc. in Biochemistry, M.Sc. in Biochemistry for Health
Workplace: Laboratory of Transgenic Models of Diseases and Czech Centre of Phenogenomics, Institute of Molecular Genetics of the Czech Academy of Sciences
Prumyslova 595, 252 50 Vestec, Czech Republic
Supervisor: Radislav Sedlacek
Co-supervisor: Eleni Papanikolaou
Project Title: Development of therapeutic exosomes and gene therapy for Diamond Blackfan Anemia (DBA)
Project Aim: In this project, we aim to develop a gene therapy treatment for Diamond-Blackfan Anemia (DBA), a condition in which the bone marrow fails to produce red blood cells, using a DBA mouse model. The aim is to develop exosomes and other delivery methods, such as lentivirus and adeno-associated virus, to correct mouse hematopoietic stem cells (HSC) using site-specific nucleases, followed by in vivo analysis of the DBA mouse model.
Expected Outcome: The expected outcome of this project is the development of therapeutic approaches based on exosomes and viral vectors for the correction of hematologic disorders, as well as the establishment of gene therapy treatment for DBA in a model system.
Contact: ana-matilde.vale@img.cas.cz
DC2 - Sabrina Capelletti
Name: Sabrina Capelletti
Nationality: Italian
Academic Background: MSc in Biotechnology and Medical Biology, Vita-Salute San Raffaele University, Milan
Workplace: Department of Molecular Cell Biology, Leiden University Medical Center
Albinusdreef 2, PO box 9600, 2300 RC Leiden, The Netherlands
Supervisor: Manuel A.F.V. Goncalves
Co-supervisor: Gerben Zondag
Project Title: Adenovector particles-mediated gene therapy of Duchenne Muscular Dystrophy
Project Aim: Investigation of chromosomal DNA cutting-free genome editing systems as advanced therapeutic approaches for Duchenne Muscular Dystrohpy (DMD)
Expected Outcome: Establishment of programmable nuclease-free genome editing tools delivered by adenoviral vector particles enabling the assessment of DMD phenotype endpoints in human muscle cell models
Contact: s.capelletti@lumc.nl
DC3 - Maryam Taghdiri
Name: Maryam Taghdiri
Nationality: Iranian
Academic Background: MSc in Clinical Immunology, Ahvaz Jundishapur University of Medical Sciences (AJUMS), Iran
Workplace: University of Freiburg medical center
Supervisor: Dr. Claudio Mussolino
Co-supervisor: Dr. Eleni Papanikolaou
Project Title: Establishing a novel system to deliver gene editing components to target cells using lipid nanoparticles with the goal of developing a new gene therapy approach for Hyper IgE syndrome
Project Aim: Testing different lipid nanoparticle formulations to establish the safe and most efficient system to deliver genome editing components to primary human hematopoietic cells (HSC) and T cells with the goal of correcting genetic mutations causing Hyper IgE syndrome (HIES)
Expected Outcome: Establishing a novel system to deliver gene editing components to target cells using lipid nanoparticles.
Contact: maryam.taghdiri@uniklinik-freiburg.de
DC4 - Aleksander Nowak
Name: Aleksander J. Nowak
Nationality: Polish
Academic Background: MSc in Pharmaceutical Biotechnology, Wrocław University of Science and Technology
Workplace: Copenhagen Center for Glycomics, Department of Cellular and Molecular Medicine
Maersk Building 07-10, Blegdamsvej 3B, DK-2200 Copenhagen N, Denmark
Supervisor: Prof. Hans H. Wandall
Co-supervisor: Dr. Roberto Nitsch
Project Title: IMGENE tool-based gene therapy of Recessive dystrophic epidermolysis bullosa (RDEB)
Project Aim: Application of enhanced CRISPR/Cas9 toolbox, developed by the IMGENE consortium, in order to in vitro treat the mutated genes in keratinocytes in the organotypic skin model, derived from the patient affected by the epidermolysis bullosa.
Expected Outcome: Delivery of novel technological and physiological insights required for the development of curative treatments for patients suffering from all types of epidermolysis bullosa.
Contact: anowak@sund.ku.dk
DC5 - Astrid Mentani
Name: Astrid Mentani
Nationality: Italian
Academic Background: I completed my bachelor’s degree in biology at the University of Bonn (Germany), where I collaborated as a Bachelor Student in the group of Prof. Sandra Blaess.
I completed my master’s degree at the Aachen University of Applied Science, while I carried out my master thesis in the Department of Neuroscience and Rare Diseases at Hoffmann-La Roche in Basel (Switzerland).
Workplace: AstraZeneca, Gothenburg, Sweden
Supervisor: Marcello Maresca
Co-supervisor: Hans H. Wandall
Project Title: Therapeutic genome editing of Cystic Fibrosis using PEn and PRINS
Project Aim: Cystic Fibrosis is a disease where the mutation of the CTFR gene leads to the build-up of thick, sticky mucus in the lungs and other organs, making it difficult to breathe and increasing the risk of infection. In this project, we aim to find a therapeutic approach for this disease, by using the latest genome editing technologies, such as Prime Editing and PRINS, that represent a more promising solution to make precise, permanent changes to the endogenous CFTR gene.
Expected Outcome: We expect to improve and optimize the latest genome editing technologies, such as Prime Editing and PRINS, to be able to use this as therapeutic approach for Cystic Fibrosis.
Contact: astrid.mentani@astrazeneca.com
DC6 - Louise Bomholtz
Name: Louise Bomholtz
Nationality: Danish
Academic Background: M.Sc. degree in Pharmaceutical Design and Engineering, The Technical University of Denmark (DTU)
Workplace: Max-Delbrück-Centrum für Molekuläre Medizin (MDC)
Supervisor: Dr. Ralf Kühn
Co-supervisor: Marcello Maresca
Project Title: Establishing gene therapy tools for IRF2BPL-related neurodevelopmental disorder using base editing and prime editing
Project Aim: Establishing base- and prime editing for the correction and restoring of heterozygous point mutations and indels in IRF2BPL-related neurodevelopmental disorder. Resulting in the generation of 3D organoids, that mimics the phenotypical characteristics of the cellular composition for the brain to determine the correct delivery method.
Expected Outcome: To increase the gene correction efficiency of patient specific mutations by the establishment and selection of a cell-specific delivery method for the delivery of the gene therapy tool of interest to the iPS-derived astrocytes and neurons. Succeeding the restoring of the IRF2BPL-related neurodevelopmental disorder with limited indel and off-target activity due to the absence of double stranded breaks.
Contact: louise.bomholtz@mdc-berlin.de
DC7 - Xiubin He
Name: Xiubin He
Nationality: Chinese
Academic Background: Master’s degree in biology (Wenzhou Medical University, China)
Workplace: University of Copenhagen, Biotech Research & Innovation Centre
Ole Maaløes Vej 5, 2200 Copenhagen, Denmark
Supervisor: Cord Brakebusch
Co-supervisor: Dr. Marcello Maresca
Project Title: Influence of chromatin modification and actin polymerization on genome editing efficiency
Project Aim: The aim of this project is to identify novel molecular targets which can improve the efficiency of gene therapy of rare diseases.
Expected Outcome: We expect to discover proteins that regulate the efficiency of gene therapy of rare diseases. Small molecule inhibitors of these proteins could then be used to improve the curative treatment of rare disease patients by genome editing.
Contact: xiubin.he@bric.ku.dk
DC8 - Aurora Giommetti
Name: Aurora Giommetti
Nationality: Italian
Academic Background: B.Sc. in Biotechnology – University of Florence, Italy (2020); M.Sc. in Medical Biotechnologies – University of Padova, Italy (2022)
Workplace: Miltenyi Biotec
Supervisor: Prof. Eleni Papanikolaou
Co-supervisor: Dr. Claudio Mussolino
Project Title: Maximizing safety and efficacy in hematopoietic stem cell (HSC) gene therapy
Project Aim: The aims of this research project are to provide further insights on the performance of the CliniMACS Prodigy® engineering system and to generate novel tools that can be used to maximize the efficacy and safety of the lentiviral transduction of Hematopoietic Stem Cells.
Expected Outcome: The expected outcome of this project is to advance our understanding of the CliniMACS Prodigy® engineering system for Hematopoietic Stem Cells (HSC). We also expect to further optimize this procedure in order to maximize the safety and the efficacy of the gene therapy applications in the field of HSC gene therapy, hopefully leading to the protocol validation.
Contact: aurorag@miltenyi.com
DC9 - Virpi Stigzelius
Name: Virpi Stigzelius
Nationality: Finnish
Academic Background: MSc and BSc in Genetics and Molecular Biosciences, University of Helsinki (Finland). Translational cancer research
Workplace: AstraZeneca R&D, Gothenburg Sweden
Supervisor: Roberto Nitsch (AstraZeneca)
Co-supervisor: Prof. Manuel Goncalves (LUMC)
Project Title: Increased safety of CRISPR-based genome editors by low immunogenicity Cas9 variants
Project Aim: Activation of the host immune response against the treatment modality is a major obstacle in therapeutic gene editing. My project aims to characterize how a key component of CRISPR-based therapies, Cas9-protein, activates the host immune response. Moreover, my aim is to mitigate the immune response activation with novel low immunogenicity Cas9 variants to improve treatment safety.
Expected Outcome: Through this project, we expect to increase the understanding of Cas9 host immunogenicity and utilize this knowledge in designing and validating low immunogenicity Cas9 variants, thus improving treatment safety.
Contact: virpi.stigzelius@astrazeneca.com
DC10 - Siang Li
Name: Siang Li
Nationality: Chinese
Academic Background: Genome editing
Workplace: University of Copenhagen, Biotech Research & Innovation Centre
Ole Maaløes Vej 5, 2200 Copenhagen, Denmark
Supervisor: Cord Brakebusch
Co-supervisor: Roberto Nitsch
Project Title: Establishment of a fluorescence reporter mouse for the determination of the efficiency of in vivo genome editing
Project Aim: We will create a novel reporter mouse for the determination of the efficiency of in vivo genome editing. Then the reporter mouse will then be used to evaluate the efficiency of different in vivo and ex vivo genome editing methods.
Expected Outcome: Using this reporter mouse we will monitor the outcome of different gene therapy techniques for the treatment of rare diseases.
Contact: Siang.li@bric.ku.dk
The large number of patients suffering from rare diseases and the insufficient availability of corresponding gene therapies in the clinic strongly illustrates the urgent requirement for more research and product development in that area.
Kick-off meeting in Copenhagen
The GetRadi consortium met in person for the first time in Copenhagen on March 30 2023. The purpose of the meeting was to let the consortium members and the fellows meet each other, network and exchange scientific ideas.
The Coordinator Cord Brakebusch and EU liaison officer Karin Norris introduced participants to the main goals of the project as well as its administrative and financial aspects.
The fellows were given an opportunity to hear about culture shock and adjustment challenges, and a lively discussion followed afterwards. The afternoon was dedicated to the presentations of the fellows' projects, with comments and input from the consortium. The day was concluded with a walk and a dinner.
Online seminar on Good Scientific Practice
On 27. April 2023, the GetRadi fellows participated in an online seminar on Good scientific practice, organized by Ralf Kühn from Max Delbrück Center.
The fellows received an introduction to the different kinds of bias that can come up in models of scientific approach, to basic principles of collaborative working and types of research misconduct. Furthermore, they became familiar with the Singapore statement on research integrity.
1st GetRadi Summer School in Leiden
On September 4-8 2023, the first GetRadi Summer school took place in Leiden. At the Summer school, the fellows presented progress of their projects and received valuable input from the consortium.
Day 2 of the Summer school was dedicated to scientific symposium “Genetic therapies: principles, models and applications” which featured poster presentations of GetRadi fellows, allowing them to receive feedback from GetRadi supervisors and local experts.
The Summer school also included sessions on outreach (provided by GetRadi partner EuroGCT), storytelling, patents and regulatory affairs as well as ethics.
1st GetRadi Winter meeting in Mölndal
On January 30-31 2024, first Winter meeting took place in Mölndal, hosted by AstraZeneca.
First day of the meeting was dedicated to updates on progress of fellow projects and Supervisory board meeting, finishing with a dinner.
On the next day, fellows and supervisors attended scientific symposium where they had an opportunity to learn about research on stem cells as well as industrial project management.
2nd GetRadi Summer school in Copenhagen
On September 2-6 2024, the second GetRadi Summer school took place in Copenhagen, organized by supervisor Hans Wandall.
At the Summer school, the fellows updated the consortium about progress of their projects and received input from everyone on project challenges they currently face.
Day 2 of the Summer school was dedicated to scientific symposium “Structure, function, and delivery of CRISPR editing tools” with invited speakers from the University of Copenhagen and Aarhus University.
The second Summer school focused on innovation - it hosted three sessions with speakers sharing their experiences with founding and running biotech companies as a researcher. The fellows were divided into groups of two, and each group developed a pitch with business proposal of their own company with topic of gene editing.
The Summer school also included sessions on outreach (provided by GetRadi partner EuroGCT), where the fellows practiced explaining their research to general audience and received a task of developing a glossary for EuroGCT website.
Online seminar on unconscious bias
On 18 December 2024, fellows participated in online seminar on Unconscious bias given by Cord Brakebusch. The fellows learned what bias is, how to become aware of unconscious bias and what strategies can be used to deal with it.
The seminar was followed by a discussion on cases of unconscious bias in hiring process and in research. Everyone shared insights from own experience on how they recognize and handle unconscious bias.
2nd GetRadi Winter meeting in Naples
The 2nd GetRadi Winter meeting in Naples took place January 14-15, 2025 kicked off with a scientific session, featuring both internal and guest speakers who will discuss various aspects of Gene Therapy Safety.
This session was also open to pre-Doc students from the PharmaTech Academy. The second day was reserved to doctoral candidates to present their research projects (closed-door meeting).
3rd GetRadi Summer School in Freiburg
3rd GetRadi Summer School took place in Freiburg, Germany,September 8-12, 2025, organized by Dr Cladio Mussolino.
Following a first day with scientific presentations by the GetRadi fellows, the next 1.5 days were covered by seminars from international speakers covering large capacity vectors, AAVs, immunological concerns of Cas9, alternative Cas9, base editing and prime editing.
Then the workshop “AI for scientific practice” was conducted for 1.5 days. The last day was dedicated to flow cytometry, CAR-T cells and HSCT.
Online seminar on Ethics in Somatic Gene Therapy
The Online Seminar by Prof. Radislav Sedlacek (IMG) on “Ethics in Somatic Gene Therapy” introduced the participants to ethical, legal, and social implications of gene therapies in preclinical development and clinical applications.
EMBO workshop “CRISPR-Cas: From Biology to Therapeutic Applications”
Dr Marcello Maresca from AZ co-organized the EMBO workshop “CRISPR-Cas: From Biology to Therapeutic Applications”, which took place in Sorrento, Italy, from November 2–5, 2025.
To this workshop, GetRadi contributed with sponsoring of a scientific session. Most GetRadi fellows took part at the workshop and presented posters.
This conference had a stellar lineup of speakers and attracted significant interest from the scientific and medical communities as well as from the pharmaceutic industry and was considered as a top meeting in genetic engineering and gene therapy in 2025.
3rd GetRadi Winter meeting in Bergisch-Gladbach
At the 3rd GetRadi Wintermeeting 16-17 February 2026, we visited the premises of Miltenyi Biotec in Bergisch-Gladbach, Germany. It was impressive to the research and development facilities and to talk to the highly motivated scientists that hosted us.
After the first day was devoted to scientific presentations of the GetRadi fellows, the second day introduced all doctoral candidates to the automated cell processors CliniMACS Prodigy, cell factories, the MACSIMA multiplex immunofluorescence staining and imaging platform, CAR-T therapies, focal molography, industrial project and portfolio management, and public funding of industrial projects.
Of note, some participants joined the Rosenmontag carnival celebrations in Cologne Monday evening.
Outreach
The European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT), https://www.eurogct.org/about-eurogct is associated partner within GetRadi.
The aim of EuroGCT is to provide understandable and accurate information about the use of cells and genetic material to treat disease. EuroGCT sources are available to general public, patients and healthcare professionals as well as to researchers.
Following projects were completed by GetRadi students with guidance of EuroGCT:
1) Glossary of terms about gene and cell therapy https://www.eurogct.org/glossary/A . The fellows have developed a glossary of terms, aiming for understandable and accessible language. This task was completed between first and second GetRadi summer school.
2) FAQ on gene and cell therapies which focuses on safety and risks. Answers to FAQ have been provided by GetRadi fellows and are available at https://www.eurogct.org/faqs.
Host: UCPH
Date: 24.04.2024
Place: Herning Gymnasium
Event: Research Day 2024: Order a researcher: Genetic engineering in medicine: Treating the untreatable or enhancing human beings?
School children participants: 25
Comments: Online talk; Introduction to chances and risks of gene editing of humans.
Host: UCPH
Date: 25.04.2024
Place: Holbæk 10. Klassecenter
Event: Research Day 2024: Order a researcher: Genetic engineering in medicine: Treating the untreatable or enhancing human beings?
School children participants: 23
Comments: In-person talk; Introduction to chances and risks of gene editing of humans
Host: UCPH
Date: 10.10.2025
Place: Panum Institute
Event: Culture Night Copenhagen 2025
School children participants: app. 50
Comments: Introduction of children and adults to different research techniques and research questions with scientific equipment and tasks.
Host: UCPH
Date: 29.01.2026
Place: Biocenter, BRIC
Event: Hands on workshop for high school students on a molecular biology subject.
School children participants: 12
Comments: 2 day introduction to CRISPR gene editing with practice.
Host: AZ
Date: 09.11.2024
Place: AstraZeneca Gothenburg
Event: AstraZeneca Family Day
School children participants: app. 90
Comments: Introduction to children how DNA is structured and how this relates to its function with hands-on work.
Host: MB
Date: 15.11.2024
Place: Miltenyi Biotec, Bergisch-Gladbach
Event: Day of the Open Door
School children participants: app. 100
Comments: Biology apprenticeship program for interested young people and schoolchildren, www.instagram.com/share/BABatwf06G
Host: MDC
Date: 22.06.2024
Place: MDC, Berlin Buch
Event: Long Night of Science
School children participants: app. 50
Comments: Oral presentation of research project of GetRadi fellow in layman terms.
Host: UKLFR
Date: 19.07.2024
Place: Gymnasium Freiburg
Event: Seminar to school children about gene editing applications for treating human disorders.
School children participants: 18
Comments: Introduction to chances and risks of gene editing of humans.
Host: IMG
Date: 08.06.2023
Place: IMG, Prague
Event: Science Fair Prague
School children participants: app. 65
Comments: Largest popular science event in the Czech Republic; Introduction to DNA structure and gene editing with hands-on work.
Host: IMG
Date: 30.05.2024
Place: IMG, Prague
Event: Science Fair Prague
School children participants: app. 70
Comments: Largest popular science event in the Czech Republic; Introduction to DNA structure and gene editing with hands-on work.
Host: LUMC
Date: 25.11.2025
Place: Albeda MBO School, Rotterdam
Event: Seminar on genome editing options in Duchenne Muscular Dystrophy in collaboration with a Duchenne patient.
School children participants: 23
Comments: Listening to the perspective of a Duchenne patient gave the school children an insight into the urgency of the GetRadi research project funded by the EU.
Host: LUMC
Date: 18.12.2025
Place: LUMC
Event: Spieren voor Spieren outreach.
School children participants: app. 40
Comments: This program raises awareness for muscle gene therapies. Here, a GetRadi fellow informed school children and adults about research techniques used in muscle disease diagnostics and general laboratory procedures. A get ready fellow https://spierenvoorspieren.nl
Contact
Professor Cord Brakebusch
Coordinator
cord.brakebusch@bric.ku.dk
Phone: +45 3532 5169
UCPH EU office
Karin Norris
Senior EU Liaison Officer
karin.norris@adm.ku.dk
Mobile: +45 2034 1987
Phone: +45 3532 6345

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Research Executive Agency.
Neither the European Union nor the granting authority can be held responsible for them.