Gene therapy and analysis of pathology in neural cell cultures: Variant infantile Batten disease

Hughes, S. M.Neverman, N. J.Peacock, H. E.Hope, K. M.Palmer, David N.Barry, Lucy A.Mitchell, Nadia L.Xu, J.2020-08-162012201220122012https://hdl.handle.net/10182/12475Mutations in CLN5 and CLN6 have been identified in three breeds of sheep. Our laboratories have developed neural cultures from these sheep to investigate early pathology and test gene therapy vectors. Gene therapy vectors are now being used to test therapeutic potental in the sheep models.Variant Infantile Batten Diseasegene therapyGene therapy and analysis of pathology in neural cell cultures: Variant infantile Batten diseaseOther