Perspectives thérapeutiques innovantes des maladies neuromusculaires

Due to a number of characteristics, therapeutic approaches of neuromuscular diseases represent complex issues. Nevertheless, they help to set the ground for many innovative breakthroughs: gene therapy by way of transfer into the target muscle or neuronal cells of full-length or minimal coding sequences, ARN “surgery” (epissotherapy or stop codon readthrough), DNA surgery, transfer or induction of compensatory genes. In parallel to these gene-based strategies, the stimulation of tissue regeneration uses embryonic or iPS stem cells that can be administered into the blood stream, with the subsequent ability of extravasation and transdifferentiation. Tissue regeneration can also be obtained by chemical stimulation of endogenous stem cells. Alternatively, various pharmacological approaches are aiming at protecting neuronal, cardiac, or skeletal muscle cells from degradation. Future therapies will probably rely on combinations of targeted gene-based and non-selective regenerative and/or cytoprotective interventions.