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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.

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News

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

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Graduate in Biomedical Sciences Gene and Cell Therapy Research Group
Start date:
25/11/2015
End date:
02/12/2015
Document: Download
Personal Tècnic Llicenciat Grup Recerca en Teràpia Gènica i Cel·lular
Start date:
24/04/2015
End date:
30/04/2015
Document: Download
Tècnic/a Ciències Biomèdiques Grup Recerca Teràpia Gènica i Cel·lular
Start date:
23/02/2015
End date:
01/03/2015
Document: Download