Postdoctoral position in combined therapy in Duchenne Muscular Dystrophy
Généthon is a nonprofit biotherapy organization supported by the French Téléthon. Its mission is the design and preclinical and clinical development of gene therapy drugs for rare diseases, mainly neuromuscular. It is an integrated R&D center making it possible to move from research to clinical development by integrating the technological innovation necessary and essential for these new therapies.
The research unit of Genethon is called INTEGRARE ("integrated genetic approaches for the treatment of rare genetic diseases", also identified as UMR_S951). Isabelle Richard's team specializes in translational research in gene therapy. Among the objectives: to overcome the limitations of systemic AAV gene delivery and apply it to the treatment of myopathies; to develop precise and safe gene editing therapeutic strategies. The team is expert in the development of gene therapy approaches for limb-girdle and Duchenne muscular dystrophies (LGMD and DMD).
In Duchenne muscular dystrophy, our lab recently characterized the perturbations of lipid metabolism (Amor et al. 2021 ; DOI: 10.1002/jcsm.12708) and molecular mechanism of mitochondrial dysfunction (Vu Hong et al. 2021a DOI: 10.4081/ejtm.2021.10012; Vu Hong et al. 2021b DOI:10.1101/2021.10.20.464950). Present studies are focused further on the molecular mechanisms of these perturbations and on the development of gene therapy and drug-based approaches for their counteraction.
The current project aims at investigating and optimizing a therapeutic approach in a mouse model for Duchenne muscular dystrophy. The approach is based on a combination of gene therapy for the restoration of dystrophin expression and a metabolic normalization of the dystrophic muscle. Under the responsibility of the PI, he/she will be responsible for designing, developing and implementing experimental protocols, working with animal models, present research results at scientific meetings, and preparing papers for publication.
Fixed-term contract of 2 years, starting no later than October 1st, 2022.
PhD: Obtained between 2019 and 2021.
Skills: Talented and motivated team-oriented scientist.
Background: Strong background in molecular and cellular biology techniques, bioinformatics skills, experience in in vitro and in vivo experiments, and a highly developed organizational sense.