Researchers at The Research Institute at Nationwide Children’s Hospital and the Ludwig Institute at the University of California, San Diego, led by Dr. Brian Kaspar and Dr. Don Cleveland, were able to show a survival benefit in both SOD1 G93A and SOD1 G37R mice by administering an adeno-associated virus serotype 9 carrying an shRNA construct directed against SOD1 (AAV9-SOD1-shRNA). The research was published online in Molecular Therapy on September 6. Female mice that were administered AAV9-SOD1-shRNA at 1 day of age showed a survival benefit of over 51.5 days or 39%. When female mice were treated at day 85, the survival benefit was still fairly impressive, 30 days or 23%. Similar results were observed in the SOD1 G37R mice, these mice showed a survival benefit of 22% when treated after disease onset. However, the team didn’t stop there; they also tested the therapy in nonhuman primates, showing that they could decrease wild-type SOD1 protein levels by 87% in the spinal cord. Although more work is still needed, the team is definitely looking to move AAV9-SOD1-shRNA into the clinic as quickly as possible. Click here to read the full story.
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