Article
Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.
Science translational medicine - 17 Nov 2021
Hagemann Tracy L, Powers Berit, Lin Ni-Hsuan, Mohamed Ahmed F, Dague Katerina L, Hannah Seth C, Bachmann Gemma, Mazur Curt, Rigo Frank, Olsen Abby L, Feany Mel B, Perng Ming-Der, Berman Robert F, Messing Albee
Abstract excerpt
Alexander disease (AxD) is a devastating leukodystrophy caused by gain-of-function mutations in GFAP, and the only available treatments are supportive. Recent advances in antisense oligonucleotide (ASO) therapy have demonstrated that transcript targeting can be a successful strategy for human neurodegenerative diseases amenable to this approach. We have previously used mouse models of AxD to show that...
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