Muscular Diseases

Human and Canine pluripotent stem cells, tools to understand and  develop new therapeutic avenues to treat Muscles Diseases.

Treatments options are currently very limited and usually symptomatic (physiotherapy, botulinum toxin, muscle relaxants, anti-inflammatory drugs) for   muscle diseases regardless of  pathology origin (genetic or acquired).  
Muscle function failure results in severe consequences for the patient : respiratory distress, impaired motility and locomotion, urinary and anal incontinence…Cytokine productions and  cell mobilisations  are central  in the maintenance of muscle functions,  the control of muscle mass and repair muscle process.

The aims of Muscles Diseases team are to determine potentials of cells derived from pluripotent cells (embryonic hES and induced pluripotent iPS stem cells) to explore new avenue to treat muscles diseases.  These strategies using human and canine cells lead to the design of disease models in a “dish“. Indeed, for muscles pathologies canine specie is particularly similar to human and therefore ideal for pre clinical studies.
The experimental reprogramming of somatic cells into iPS cells (induced pluripotent cells) with the essential characteristics of embryonic stem cells (ES) (unlimited self renewal capacity and differentiation into  all cell types forming an organism) is performed on normal and mutant human and canine somatic cells. The pathways of differentiation into muscle progenitor cells will be define using an automated combinatorial approach.
Cellular tools will enable the exploration of patient-oriented disease investigation, in parallel with gene correction and cell transplantation strategies.  Therapeutically relevant molecules will be identified using high throughput screening technologies (HTS).