Antisense therapy in a new rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.
Tracy L. Hagemann, Berit E. 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
This paper introduces a new rat model of Alexander disease that exhibits key pathological features and motor impairment, demonstrating that antisense oligonucleotide therapy can reverse GFAP pathology and white matter deficits.
Science Translational Medicine · Nov 17, 2021Peer-reviewedNeuroscience