Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment

著者: Tracy L. Hagemann, Berit Powers, Ni‐Hsuan Lin, Ahmed F. Mohamed, Katerina L. Dague, Seth C. Hannah, Gemma Bachmann, Curt Mazur, Frank Rigo, Abby L. Olsen, Mel Β. Feany, Ming‐Der Perng, Robert F. Berman, Albee Messing - Science Translational Medicine 2021 被引用: 55

-targeted ASO provides long-lasting suppression, reverses GFAP pathology, and, depending on age of treatment, prevents or mitigates white matter deficits and motor impairment. In this report, we characterize an improved animal model of AxD with myelin pathology and motor impairment, recapitulating prominent features of the human disease, and use this model to show that ASO therapy has the potential to not only prevent but also reverse many aspects of disease.