Animal Physiopathology and Biotherapy of the muscle and nervous system- PAnTher

Director - Professor Marie-Anne COLLE

Contact : marie-anne.colle(at)

Co-director - Doctor Karl ROUGER

Contact : karl.rouger(at)

Key words

Animal model / Muscle / Nervous system / Animal Physiopathology / Cell therapy / Gene therapy / Translational research

Positioning and thematics of the Unit


In terms of high scientific orientation and positioning, with a scientific focus on biomedical research and intelligence action on emerging or re-emerging diseases, the UMR is part of “One World, One Health” initiative, promoted from 2004 by WCS (Wildlife Conservation Society) and whose strategy is based on the analysis of the interdependence of the health of human and animal populations. This initiative promotes interdisciplinarity in worlwide communication and collaborations related to human health care as well as animal health care.


Cell and gene therapies of Duchenne Muscular Dystrophy (DMD)

  • The research works of the UMR on the isolation and characterization of therapeutic somatic stem cells from muscle (MuStem) show major and long term clinical improvement of the relevant model of DMD, the GRMD dog. They represent a major scientific breakthrough in transitional research and provide opportunities for treatment of the most common human genetic disease. In continuity,the UMR develops a research program on the use of MuStem cells as innovative drug.

    The UMR is helping to develop another clinical product for the DMD treatment, a AAV8 carrying a U7 snRNA specific for the exon skipping of the dystrophin transcript, administered  by intravenous locoregional injection.

Gene therapy of Central Nervous System (CNS)

  • In addition to collaborative preclinical results obtained by intracerebral gene therapy in MPSI dog, MPSIIIB dog and in non-human primates, and that will lead to two phase I / II clinical trials, the UMR develops a research program dedicated to intrathecal gene transfer in the non-human primate and to correcting neurological manifestations of glycogen storage disease type II (Pompe disease).

The platform of expertise in Pathology (APEX)

The need for specific skills in anatomical pathology in biomedical research has led to the creation of a platform of expertise in anatomical pathology called APEX. Its originality is to develop synergies between the specific skills of the partner teams and expertise in animal physiopathology. It also provides support missions, training and development. It is integrated into the platform "Animal biotherapy and physiopathology", labeled by the GIS IBiSA in December 2008.


Website APEX