eCORE
- Advanced Therapies and Advanced Interventions, Nanomedicine, Transplant & Donation
Team
Group Leader
Jordi Barquinero Mañez
Principal Investigator (PI)
Jordi Barquinero Máñez
Researchers
María Pallarés Masmitjà
PhD Students
Rocío Piñera Moreno
Selected Publications
Hubert Smeets, Bram Verbrugge, Xavier Bulbena, Liliya Hristova, Julia Vogt, Isabelle van Beckhoven; all participants; Patient organizations/patient representatives: LAMA2-Europe, Voor Sara-the Netherlands, ImpulsaT-Spain, LAMA2-France, CMD-Turkey, LAMA2-Bulgaria, Cure-CMD
European Joint Programme on Rare Diseases workshop: LAMA2-muscular dystrophy: paving the road to therapy March 17-19, 2023, Barcelona, Spain
Neuromuscul Disord . 2024 Mar:36:16-22
DOI: 10.1016/j.nmd.2024.01.001
IF: 2.7
Paula Río, Josune Zubicaray, Susana Navarro, Eva Gálvez, Rebeca Sánchez-Domínguez, Eileen Nicoletti, Elena Sebastián, Michael Rothe, Roser Pujol, Massimo Bogliolo, Philipp John-Neek, Antonella Lucía Bastone, Axel Schambach, Wei Wang, Manfred Schmidt, Lise Larcher, José C Segovia, Rosa M Yáñez, Omaira Alberquilla, Begoña Díez, María Fernández-García, Laura García-García, Manuel Ramírez, Anne Galy, Francois Lefrere, Marina Cavazzana, Thierry Leblanc, Nagore García de Andoin, Ricardo López-Almaraz, Albert Catalá, Jordi Barquinero, Sandra Rodríguez-Perales, Gayatri Rao, Jordi Surrallés, Jean Soulier, Cristina Díaz-de-Heredia, Jonathan D Schwartz, Julián Sevilla, Juan A Bueren; FANCOLEN-1 gene therapy investigators
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
2025 Dec 21;404(10471):2584-2592. Epub 2024 Dec 3.
DOI: 10.1016/S0140-6736(24)01880-4
IF: 98.4
Selected Projects
Congenital muscular dystrophy type 1A: fibrosis inhibition and correction through gene editing.
Principal Investigator: Francina Munell & Jordi Barquinero
Agency: Instituto de Salud Carlos III (PI22/01027)
Funding: 117,370 €
Period: 2023-2025
Development of a novel gene therapy strategy and adaptation of a patient cohort for clinical trials in merosin-deficient congenital muscular dystrophy
Principal Investigator: Francina Munell & Jordi Barquinero
Agency: Instituto de Salud Carlos III (PI19/00295)
Funding: 139,150 €
Period: 2020-2024 (extended)
Towards a gene therapy for MDC1A
Principal Investigator: Francina Munell & Jordi Barquinero
Agency: Ajuntament de Castellbisbal / Associació Impulsa't
Funding: 20,000 €
Period: 2024
Patents
An in vitro method for transducing a target cell
Priority Number: EP 24382581.7
Priority Date: 30/05/2024
Applicants: 45% VHIR, 35% UAB, 20% UdL
Selected Dissemination Activities
Gene therapy: A dream come true. Workshop "21st Century Medicine: The Path to the Future
Place: La Casa Elizalde (Barcelona)
Date: 01/01/1970
Brief: Conference about gene therapy for a general audience
Applications of Gene Therapy in medicine. Information pills of gene therapy in hemophilia.
Place: Organized by the Spanish Society of Thrombosis and Haemostasis (SETH). Online activity.
Date: 26/07/2025
Brief: Virtual short class on gene therapy for hemophilia
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The human pandemics (editorial)
Place: Journal "Treballs de la Societat Catalana de Biologia", Vol 74
Date: 26/07/2025
Brief: An editorial written by Heribert Playa and Jordi Barquinero in the annual volume of this journal. Also, JB is the Publication officer of this Academic Society.
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