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Autor:
Berit Powers, Albee Messing, Ahmed F. Mohamed, Tracy L. Hagemann, Curt Mazur, Robert F. Berman, Seth C. Hannah, Ni-Hsuan Lin, Katerina L. Dague, Mel B. Feany, Abby L. Olsen, Frank Rigo, Ming-Der Perng, Gemma Bachmann
Publikováno v:
Sci Transl Med
Science translational medicine, vol 13, iss 620
Science translational medicine, vol 13, iss 620
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 targe