Chinese researchers have revived a therapeutic technique designed to over-express utrophin, an endogenous protein very similar to dystrophin:
- they used a genome-editing approach in several models (cellular and animal) of Duchenne muscular dystrophy (DMD) combined with a Myo-AAV muscle-specific viral vector (to obtain MyoAAV-UA).
- after administration, robust and long-lasting overexpression of utrophin was obtained in the different models, without any particular toxicity problems, including in primates,
- a real functional benefit was observed.
This work is highly attractive and revives the interest in this substitutive approach in DMD, which is a priori better tolerated because it is less immunogenic.
