Beijing researchers report the results of preclinical studies on a new-generation gene therapy for SMA:
- the new transgene (AAV9-coSMN1) contains an optimised codon, carried by an adeno-associated virus of the AAV9 type,
- mice from the Taiwanese model and non-human SMA primates were used and studied after intrathecal injection of the transgene,
- this modification in the composition of the transgene led to an increase in the production of the SMN1 protein in vitro and an improvement in the functional and histological parameters of the motor neurons, with no reported tolerance problems.
Clinical studies are already underway with the same product developed and manufactured in China (OptimumGene®).
