NKH Research Updates

Gene therapy exploring vector production and safety studies, and RNA treatment study launched! Highlights: Overall strategy The UCL research strategy continues to focus on two main themes: They aim (i) to better understand theNKH disease process, and (ii) to use this information to develop new treatments and evaluate howwell they work. Understanding NKH – experimental […]

UCL Updated Research Paper on NKH Gene therapy and AMT gene therapy research started Highlights: – Creating more complex GLDC mouse models– AMT work has started, both in growing cell models, and in gene therapy research– UCL has identified metabolic alterations that were previously unrecognised in children with NKH (research paper on it’s way)– Cinnamate

Discovery of two compounds that may impact glycine levels in the brain. Overview