French researchers, in collaboration with the Généthon laboratory, report the results of a study conducted on rats with Duchenne muscular dystrophy (DMD) treated with microdystrophin gene therapy:
- this work comes in the context of toxicity observed in some DMD patients who received microdystrophin using an AAV viral vector;
- a rat optimised to best mimic DMD (DMDmdx) received injections of the transgene at various doses, compared to healthy rats,
- the rats treated with the highest dose experienced a significant number of sudden deaths, possibly related to cardiac arrhythmias.
Although it remains difficult to draw a formal parallel with the therapeutic trials currently underway, the authors stress the need for caution with this type of AAV-mediated gene therapy, especially at high doses.
