New research in animals by scientists at Washington University School of Medicine in St. Louis and the Roslin Institute at the University of Edinburgh in Scotland suggests enzyme replacement therapy may slow brain degeneration. The Washington University researchers evaluated the therapy in mice, and researchers in Scotland evaluated the treatment in a sheep model of the disease. The findings underscore potential treatments for the genetic condition, also known as CLN1 disease.
At the beginning of April we received a notice of award of a new NIH R21 grant from NINDS. This is to explore the extent of peripheral nerve disease in CLN1 mice and treat this via gene therapy. This work will be done together with long term collaborator Mark Sands (Washington University, Medicine) and Alison […]
Keigo Takahashi’s review article on “Glial dysfunction and its contribution to the pathogenesis of the neuronal ceroid lipofuscinoses” was just accepted by Frontiers in Neurology. Keigo’s pre-print (below) on the characterization of Cln2 mice revealing their seizure phenotype and the pathological involvement of interneurons is also now available on BioRxiv. We are just completing another […]