Gene therapy for SOD1-linked ALS

What is it about?

This work shows that treating ALS mice with a gene therapy vector that silences the SOD1 gene delays disease onset and prolongs their survival. It also shows that while non-treated mice, like ALS patients, show a decrease in the respiratory function, treated mice maintain their breathing. Finally, when we made the mice run on a rotating rod, the untreated mice failed the test rather quickly, while the treated mice maintain their motor function longer. Importantly, it's the first time that gene therapy has been showed to improve respiratory and motor functions. An important factor is the age at which the mice are treated. A lot of research has been done in pre-symptomatic ALS mice. However, here we have treated post-symptomatic adult mice, which mimics closely when the treatment would be administered to patients. Besides the work done with mice, we also tested the gene therapy vector in small monkeys, to see whether we can treat the entire spinal cord with only one injection. This work shows that not only we can treat the entire spinal cord, but the treatment is safe, as the monkeys did not show any negative side-effects.

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Why is it important?

This work shows that even when treating post-symptomatic adult mice, gene therapy can have an impact on the quality of life of the animals (mobility, breathing) and their overall survival. This work also shows that this gene therapy treatment is safe and efficient in monkeys. Both of these aspects have to be carefully considered before treating patients.