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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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Maria Pallares Masmitja

Maria Pallares Masmitja

Postdoctoral researcher
Gene and Cell Therapy
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Oscar Garriga Monterde

Oscar Garriga Monterde

Research technician
Gene and Cell Therapy
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Sofía Travers Rama

Sofía Travers Rama

Predoctoral researcher
Gene and Cell Therapy
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Rocío Piñera Moreno

Rocío Piñera Moreno

Research technician
Gene and Cell Therapy
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Jordi Barquinero Mañez

Jordi Barquinero Mañez

Head of group
Gene and Cell Therapy
Read more
Maria Pallares Masmitja

Maria Pallares Masmitja

Postdoctoral researcher
Gene and Cell Therapy
Read more
Oscar Garriga Monterde

Oscar Garriga Monterde

Research technician
Gene and Cell Therapy
Read more
Sofía Travers Rama

Sofía Travers Rama

Predoctoral researcher
Gene and Cell Therapy
Read more
Rocío Piñera Moreno

Rocío Piñera Moreno

Research technician
Gene and Cell Therapy
Read more

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: -

Thesis

Caracterización funcional de una nueva familia de péptidos bioactivos derivados de filamentos intermedios y estudio de sus posibles aplicaciones en salud humana

PhD student: Rocío Piñera Moreno
Director/s: Jordi Barquinero Mañez, Lluis Tort Bardolet
University: Universitat Autònoma de Barcelona
Year: 2024

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.