Two weeks ago, uniQure released a safety update for the first 10 participants in the low-dose cohort of the phase I/II trial of AMT-130, the first experimental gene therapy to be tested in people with Huntington’s disease. The editors of HD Buzz recently sat down with Dr. Ricardo Dolmetsch, President of Research and Development at uniQure, to unpack the press release and get some clarity on what’s next for AMT-130. For the full update, visit HDBuzz.
Gut health may contribute to onset of HD
Several neurodegenerative disorders have been linked with changes in gut health. A group of researchers based in California, including former HDSA Human Biology Fellow, Dr. Ali Khoshnan, set out to determine whether the same is true for Huntington’s disease. The study tested the effects of damaging gut bacteria on HD symptoms in fruit flies. The results suggest that the introduction of harmful bacteria increases aggregation of huntingtin protein, worsens movement impairment, and reduces lifespan in HD flies. They also found that treating gut dysbiosis delays HD progression in fruit flies. The notion that gut bacteria may alter the toxicity of huntingtin protein and affect the progression of HD is a novel finding that may have implications for future gut-based interventions for HD. To read the full scientific article, click here.
Beyond the HD Gene: Research Webinar from HDF
The Hereditary Disease Foundation Research Spotlight Webinar series features scientists working to identify treatments for Huntington’s disease and other brain disorders, who present their work in plain language. Check out the HDF’s most recent webinar, presented by Dr. Steve Finkbeiner and Dr. Leslie Thompson, which focused on therapeutic research into genes that interact with huntingtin. Click here to watch.