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Gene and Cell Therapy

One of our current research goals is to develop a gene editing strategy that combines viral and non-viral vectors to treat Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE), a rare disease due to inactivating mutations in the gene encoding thymidine phosphorylase, which has already demonstrated therapeutic efficacy in an animal model of the disease.

On the other hand, in collaboration with the Neuropediatrics group (Dra. F. Munell) and that of Dr. R. Hernández (University of Navarra), we are investigating another gene therapy approach based on high-capacity adenoviral vectors using a murine model of congenital muscular dystrophy type 1A (CMD1A).

Finally, in collaboration with the group of Dr. J. Verdaguer (University of Lleida), we are studying the mechanism of action, the cytolytic and pro-inflammatory activities, as well as several biomedical applications of a new family of peptides derived from intermediate filaments that were discovered by chance.

Team

Jordi Barquinero Mañez

Jordi Barquinero Mañez

Head of group
Gene and Cell Therapy
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Rocío Piñera Moreno

Rocío Piñera Moreno

Research assistant
Gene and Cell Therapy
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Jordi Barquinero Mañez

Jordi Barquinero Mañez

Head of group
Gene and Cell Therapy
Read more
Rocío Piñera Moreno

Rocío Piñera Moreno

Research assistant
Gene and Cell Therapy
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Research lines

Gene therapy of congenital muscular dystrophy due to merosin deficiency

Development of a new gene therapy strategy based on high-capacity adenoviral vectors, which we carry out in collaboration with the group of Dr. Rubén Hernández, from the University of Navarra.

IP: Jordi Barquinero Mañez

Preclinical studies on gene therapy for Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE)

IP: -

Tolerance induction by hematopoietic gene therapy targeting myeloid cells in a murine model of autoimmunity (EAE)

IP: -

Projects

Teràpia gènica en MDC1A

IP: Jordi Barquinero Mañez
Collaborators: Laia Perez Lasarte, Maria Pallares Masmitja
Funding agency: Ministerio de Ciencia e Innovación-MICINN
Funding: 67400
Reference: JDC2022-049508-I
Duration: 01/01/2024 - 31/12/2025

Ministerio de Ciencia

Fisiologia i fisiopatologia digestiva

IP: Chaysavanh Manichanh
Collaborators: Itxarone Izaskun Bilbao Aguirre, Zaida Soler Luque, Jordi Barquinero Mañez, Oscar Segarra Canton, Gerard Serrano Gomez, Cristina Dopazo Taboada, Rocío Piñera Moreno
Funding agency: Agència Gestió Ajuts Universitaris i de Recerca
Funding: 40000
Reference: 2021 SGR 00459
Duration: 01/01/2022 - 30/06/2025

Distrofia muscular congénita tipo 1A: inhibición de la fibrosis y corrección mediante edición génica

IP: Jordi Barquinero Mañez
Collaborators: Laura Costa Comellas, Francina Munell Casadesus, Gómez Andrés, David, Rocío Piñera Moreno, Maria Pallares Masmitja
Funding agency: Instituto de Salud Carlos III
Funding: 117370
Reference: PI22/01027
Duration: 01/01/2023 - 31/12/2025

Desarrollo de una nueva estrategia de terapia génica y adecuación de una cohorte de pacientes para ensayos clínicos en distrofia muscular congénita por déficit de merosina

IP: Jordi Barquinero Mañez
Collaborators: Francina Munell Casadesus, Esther Toro Tamargo
Funding agency: Instituto de Salud Carlos III
Funding: 139150
Reference: PI19/00295
Duration: 01/01/2020 - 30/06/2024

Publications

Absence of p.R50X Pygm read-through in McArdle disease cellular models.

PMID: 31848135
Journal: Disease Models & Mechanisms
Year: 2020
Reference: Dis Model Mech. 2020 Jan 13;13(1). pii: dmm.043281. doi: 10.1242/dmm.043281.
Impact factor: 4.651
Publication type: Paper in international publication
Authors: Tarraso, Guillermo, Real-Martinez, Alberto, Pares, Marta, Romero-Cortadellas, Lidia, Puigros, Laura, de Luna, Noemi, Brull, Astrid, Martin, Miguel Angel, Arenas, Joaquin, Lucia, Alejandro et al.
DOI: 10.1242/dmm.043281

Myeloid-derived suppressor cells can be efficiently generated from human hematopoietic progenitors and peripheral blood monocytes.

PMID: 28108746
Journal: IMMUNOLOGY AND CELL BIOLOGY
Year: 2017
Reference: Immunol Cell Biol. 2017 Jul;95(6):538-548. doi: 10.1038/icb.2017.4. Epub 2017 Jan 21.
Impact factor: 4.557
Publication type: Paper in international publication
Authors: Casacuberta-Serra, Silvia, Pares, Marta, Golbano, Arantxa, Coves, Elisabet, Espejo, Carmen, Barquinero, Jordi et al.
DOI: 10.1038/icb.2017.4

Thesis

Desenvolupament de noves estratègies terapéutiques per les distròfies musculars

PhD student: Penelope Romero Duque
Director/s: Francina Munell Casadesus, Jordi Barquinero Mañez
University: Universidad Autònoma de Barcelona
Year: 2022

Preclinical assessment of a gene editing approach for MNGIE

PhD student: Marta Parés Casellas
Director/s: Jordi Barquinero Mañez
University: Universidad Autònoma de Barcelona
Year: 2020

Antigen-specific MDSCs induce immunological tolerance in an experimental model of multiple sclerosis. Generation of human MDSCs from hematopoietic progenitors as a therapeutic tool

PhD student: Silvia Casacuberta Serra
Director/s: Jordi Barquinero Mañez, Carmen Espejo Ruiz
University: Universidad Autònoma de Barcelona
Year: 2016

Estudio de los mecanismos terapéuticos en la inducción de tolerancia inmunológica en un modelo animal de esclerosis múltiple

PhD student: Alba Gomez Morago
Director/s: Jordi Barquinero Mañez, Carmen Espejo Ruiz
University: Universidad Autònoma de Barcelona
Year: 2012

Estudios preclínicos de terapia génica basada en células madre hematopoyéticas para el MNGIE

PhD student: Javier Torres Torronteras
Director/s: Ramon Martí Seves, Jordi Barquinero Mañez
University: Universidad Autònoma de Barcelona
Year: 2011

Blog

News

Over the course of two days, experts presented the latest advances in vectors, different gene modification techniques and their transfer to clinical practice.