The lab develops gene therapy approaches to treat the Duchenne muscular dystrophy (DMD). DMD affects 1 in 5000 new-born males and is one of the most common recessive disorders in the human population.
The disease is caused by mutations in the dystrophin gene. In the absence of the dystrophin protein, muscle strength, flexibility and stability are lost, DMD patients are restricted to wheelchair by the age of 12 years and usually succumb in their second to fourth decade of life.
Despite exhaustive clinical management and considerable progress in gene-based, cell-based and pharmacological strategies, there is currently no effective treatment for DMD.
Recently, gene therapy using recombinant adeno-associated virus (rAAV) to deliver a truncated but partially functional micro-dystrophin gene entered in clinical trials bringing hope to the field.
Our project consists in developing next generation gene therapy approaches for DMD. Based on surrogate genes able to compensate for the lack of dystrophin in DMD, we aim to develop innovative gene therapies applicable to all DMD patients as a robust alternative to the traditional micro-dystrophin strategies.
We will focus on two different approaches : a gene replacement strategy using newly optimized surrogate micro-genes; a CRISPR / Cas9 based strategy to upregulate the expression of the surrogate endogenous genes.
The candidate will participate in testing some aspects on these strategies in human DMD myoblasts and murine DMD model.
Dès que possible