AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model.

Both vectors lead to a highly significant extension of survival and amelioration of the motor phenotype if compared to the empty vector (null), confirming the efficacy of gene therapy in the SMARD1 mice.

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Conversely, in the P546-treated group, there was a clear trend toward reduced fibrosis; notably, the difference between wild-type and P546 was no longer statistically significant, indicating a recovery of fibrosis levels toward those of wild-type mice (Fig. 3 F, G).

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