NeuroscienceMolecular TherapyPeer-reviewed
Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy
Sunetra Sase, Julia Liao Hacker, Prabhat R. Napit…·Adeline L. Vanderver
This study shows that suppressing Tubb4a with an ASO improves symptoms and extends lifespan in a mouse model of H-ABC leukodystrophy.
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