Gene Therapy for treatment of rare inherited Arrhythmogenic Cardiomyopathy

Informazioni relative al progetto

GEREMY

ID dell’accordo di sovvenzione: 101080204

DOI

10.3030/101080204

Data della firma CE 7 Aprile 2023

Data di avvio 1 Maggio 2023

Data di completamento 30 Aprile 2027

Finanziato da

• Health

Costo totale

€ 7 774 103,75

Contributo UE

€ 7 774 103,75

Coordinato da

STICHTING NETHERLANDS HEART INSTITUTE
Netherlands

CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.

I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .

Risultati finali

Data Management Plan (1)

Data Management Plan

A data management plan (DMP) will describe how the data will be managed, collected, stored, what standards will be used, and how data will be handled and protected during and after the completion of the project.

Websites, patent fillings, videos etc. (1)

Project Website

We will generate a Project Website to support our Communication and dissemination activities.

Pubblicazioni

Peer reviewed articles (11)

Plasma proteomics stratification identifies phospholamban R14del carriers at risk for disease progression

Autori: Frederik E Deiman, Taner Arslan, Remco de Brouwer, Ian Henry, Lars Löfgren, Anders Cavallin, Filippos Michopoulos, Ralf Nilsson, Marcus Henricsson, Christine Ahlstrom, Pia Davidsson, Nils Bomer, Niels Grote Beverborg, Peter van der Meer
Pubblicato in: Cardiovascular Research, Numero 122, 2026, ISSN 0008-6363
Editore: Oxford University Press (OUP)
DOI: 10.1093/CVR/CVAG089

The PLN Foundation is striving for a cure, but who owns the disease?

Autori: Luuk Kerckhaert, Pieter Glijnis, Annet N. Linders, Cor Oosterwijk, Pieter A. Doevendans
Pubblicato in: Netherlands Heart Journal, Numero 33, 2025, ISSN 1568-5888
Editore: Springer Science and Business Media LLC
DOI: 10.1007/S12471-025-01961-4

Tools and tactics for studying alternative splicing

Autori: Rui Sousa-Luís, Maria Carmo-Fonseca
Pubblicato in: Nature Reviews Genetics, 2026, ISSN 1471-0056
Editore: Springer Science and Business Media LLC
DOI: 10.1038/S41576-026-00952-4

Moral Attitudes on Somatic Gene Editing for Inherited Cardiomyopathy: A Qualitative Interview Study with Key Stakeholders in the Netherlands

Autori: Jannieke N. Simons, Phaedra Locquet, Pascal Borry, Johannes J. M. van Delden, Rieke van der Graaf
Pubblicato in: Journal of Bioethical Inquiry, 2026, ISSN 1176-7529
Editore: Springer Science and Business Media LLC
DOI: 10.1007/S11673-026-10565-5

Phosphoproteomics distinguishes disease-specific mechanisms for human phospholamban cardiomyopathy reversible by RNA therapy

Autori: Frederik E. Deiman, Nils Bömer, Pia Davidsson, Daniela Später, Annet N. Linders, Itamar B. Dias, Karla F. Arevalo Gomez, Antonio Esquivel Gaytan, Jumo Zhu, Anna Walentinsson, Susanna Engberg, Neil Hattersley, Damla Etal, Christine Ahlstrom, Adam E. Mullick, Kenny M. Hansson, Herman H. W. Silljé, Niels Grote Beverborg, Peter van der Meer
Pubblicato in: Signal Transduction and Targeted Therapy, Numero 11, 2026, ISSN 2059-3635
Editore: Springer Science and Business Media LLC
DOI: 10.1038/S41392-026-02791-5

Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy

Autori: Eirini Kyriakopoulou, Danielle Versteeg, Hesther de Ruiter, Ilaria Perini, Fitzwilliam Seibertz, Yannic Döring, Lorena Zentilin, Hoyee Tsui, Sebastiaan J. van Kampen, Malte Tiburcy, Tim Meyer, Niels Voigt, van J. Peter Tintelen, Wolfram H. Zimmermann, Mauro Giacca, Eva van Rooij
Pubblicato in: Nature Cardiovascular Research, Numero 2, 2026, ISSN 2731-0590
Editore: Springer Science and Business Media LLC
DOI: 10.1038/S44161-023-00378-9

Disparities in access to gene therapy in the European Union: ethical and regulatory challenges

Autori: Margaux Reckelbus, Riya Mohan, Phaedra Locquet, Eva Van Steijvoort, Isabelle Huys, Pascal Borry
Pubblicato in: Journal of Law and the Biosciences, Numero 12, 2025, ISSN 2053-9711
Editore: Oxford University Press (OUP)
DOI: 10.1093/JLB/LSAF018

Unmet health-related needs of heritable arrhythmogenic cardiomyopathy carriers in Belgium: The UR-HEART survey study

Autori: Phaedra Locquet, Eva Van Steijvoort, Pascal Borry, Zilke Claessens, Margaux Reckelbus, Tomas Robyns, Isabelle Huys
Pubblicato in: Heart Rhythm O2, Numero 6, 2026, ISSN 2666-5018
Editore: Elsevier BV
DOI: 10.1016/J.HROO.2025.08.029

Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges

Autori: Seyed Younes Hosseini, Rahul Mallick, Petri Mäkinen, Seppo Ylä-Herttuala
Pubblicato in: Human Gene Therapy, Numero 35, 2024, ISSN 1043-0342
Editore: Mary Ann Liebert Inc
DOI: 10.1089/hum.2024.043

Public perception and willingness to accept somatic gene therapy: A Belgian survey study

Autori: Phaedra Locquet, Margaux Reckelbus, Eva Van Steijvoort, Pascal Borry, Bram Korbmacher, Sofie Gordts, Lauren Vanceer, Isabelle Huys
Pubblicato in: Open Research Europe, Numero 5, 2026, ISSN 2732-5121
Editore: F1000 Research Ltd
DOI: 10.12688/OPENRESEUROPE.21462.2

Gene Therapy for Heart Failure

Autori: Hanna Wang, Anni Määttä, Seppo Ylä-Herttuala
Pubblicato in: Journal of Cardiovascular Translational Research, Numero 19, 2026, ISSN 1937-5387
Editore: Springer Science and Business Media LLC
DOI: 10.1007/S12265-025-10736-6