Skip to main content

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.

Publications

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

Myeloid-derived suppressor cells are generated during retroviral transduction of murine bone marrow.

PMID: 23192196
Journal: CELL TRANSPLANTATION
Year: 2014
Reference: Cell Transplant. 2014 Jan;23(1):73-85. doi: 10.3727/096368912X658971. Epub 2012 Nov 27.
Impact factor:
Publication type: Paper in international publication
Authors: Barquinero, Jordi; Casacuberta-Serra, Silvia; Eixarch, Herena; Espejo, Carmen; Gimeno, Ramon; Gomez, Alba; Lopez-Estevez1, Sergio; Mansilla, Maria Jose; Montalban, Xavier; Pereira, Sonia et al.
DOI: 10.3727/096368912X658971

Up-regulation of inducible heat shock protein-70 expression in multiple sclerosis patients.

PMID: 24328534
Journal: AUTOIMMUNITY
Year: 2014
Reference: Autoimmunity. 2014 Mar;47(2):127-33. doi: 10.3109/08916934.2013.866104. Epub 2013 Dec 12.
Impact factor:
Publication type: Paper in international publication
Authors: Castillo, Joaquin; Castillo, Mireia; Comabella, Manuel; Espejo, Carmen; Mansilla, Maria Jose; Martin, Roland; Montalban, Xavier; Rio, Jordi et al.
DOI: 10.3109/08916934.2013.866104

Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE.

PMID: 24448160
Journal: MOLECULAR THERAPY
Year: 2014
Reference: Mol Ther. 2014 May;22(5):901-7. doi: 10.1038/mt.2014.6. Epub 2014 Jan 22.
Impact factor:
Publication type: Paper in international publication
Authors: Auricchio, Alberto; Barquinero, Jordi; Cabrera-Perez, Raquel; Camara, Yolanda; Di Meo, Ivano; Hirano, Michio; Marti, Ramon; Pizzorno, Giuseppe; Torres-Torronteras, Javier; Viscomi, Carlo et al.
DOI: 10.1038/mt.2014.6

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.