Fixed term contract for 2 years

Title: Identification of modifiers of the dystrophin gene expression to restore functional
Proteins using HTS technology on cell progenies derived from human induced pluripotent stem cells
I-stem, the Institute for Stem cell Therapy and Exploration of Monogenic diseases, established on 1st January 2005, is a research and development centre dedicated to the treatment based on the potential offered by pluripotent stem cells applicable to rare diseases of genetic origin. Programs of each of the research teams are devoted either to a set of genetic diseases or the development of new technologies. Currently, the major pathological indications studied concern diseases of muscle, neurons, skin, retina, neurodegenerative diseases and those associated with developmental abnormalities of the cerebral cortex or neural crest and diseases characterized by accelerated aging. Research teams explore technological tools, especially cells production in bulk and screening of chemical compounds. Research teams permanently interact with each other around common projects around the technological platforms that are shared.
The successful candidate will work within the muscle disease team with the objectives of opening therapeutic avenues for muscle genetic diseases, such as Duchenne Muscular Dystrophy caused by lesion in dystrophin gene, using cell derived from human pluripotent stem cells (hES, IPS derived from healthy patients and from DMD patients). Recently, the Muscular disease team, led by Christian Pinset, has developed a cell system to express of human dystrophin in cell derived from human pluripotent stem cells. The simplicity and robustness of this cell system allow the development of high throughput screening approaches (HTS). With hIPS cells derived from DMD patients with different genetic lesions, the objective of the project will be the identification of modifiers of dystrophin (exon skipping to restore a genetic reading frame, suppression translation termination at in frame premature termination expression) to restore the dystrophin functionality using HTS approaches.

Keywords: hES, IPS, HTS, Dystrophin, Developmental and Cell biology, Duchenne
Myopathy
Application
The candidate should be creative, have a background in developmental biology, he/she must have an interest in translational medicine and automated approaches. Please send to cpinset@istem.fr a detailed CV, including a complete bibliography and recommendation letters.
This fellowship is dedicated to candidates trained in France (University degree or PhD) and curently having a postdoctoral position in other country.

Location : I-Stem, Evry, 30 km south of Paris, France

Contact Christian Pinset cpinset@istem.fr 33664784433
Further informations : http://www.istem.eu/en/biotherapies/maladies-musculaires/…