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A three-drug combination worked better than any of them alone in treating ALS caused by mutations in the SOD1 gene, per a new ...
Dokholyan explained that SOD1 typically exists as a dimer, a protein composed of two identical units. Under certain conditions, SOD1 will change its shape and reassemble itself into a three-unit ...
Building on the work of the late Professor Justin Yerbury, who passed away from MND in 2023, his research team found that a combination of three drugs could be used to target the SOD1 protein ...
In the study, chemist Jeff Agar and his colleagues researched mutations in the gene that makes a particular protein, known as SOD1, responsible for causing much of the familial form of ALS. Genetic ...
Wild-type SOD1 is among the most stably-folded intracellular proteins known, retaining significant conformation and enzymatic activity in the presence of strong protein denaturants. With these ...
Over 20 percent of patients with ALS have a mutation in a protein called superoxide dismutase 1 (SOD1). 15 This mutation causes SOD1 to bind and block an anti-apoptotic protein, B cell lymphoma-2 (BCL ...
Most adverse events were mild and transient. Significant target engagement: Treatment with RAG-17 led to a substantial reduction of SOD1 protein levels in the cerebrospinal fluid (CSF), exceeding ...
By reducing the production of the toxic mutant SOD1 protein, RAG-17 aims to slow or halt the progression of SOD1-ALS. Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease ...