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CMS – Ricardo Maselli, M.D.

MDA Admin
CMS - Ricardo Maselli, M.D.
Ricardo Maselli says his team’s goal is to implement stem cell-based gene therapy in patients with endplate AChE deficiency after preclinical studies in animals are completed. They hope to be able to treat patients with endplate deficiency of AChE and other forms of congenital myasthenic disorders with this novel form of cell-based molecular therapy.

Ricardo Maselli, a professor in the neurology department at the University of California Davis was awarded an MDA research grant totaling $300,000 over three years to test whether transplantation of stem cells engineered to secrete a needed protein could be a beneficial treatment strategy in congenital myasthenic syndromes (CMS). If studies in a mouse model are successful, testing could advance to patients. Promising results could steer the field to a new therapeutic strategy for CMS.

Funding for this MDA research grant began Aug. 1, 2015.