Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy
Sunetra Sase, Julia Liao Hacker, Prabhat R. Napit, Anjali Bhagavatula, Sarah Woidill, Annemarie D’Alessandro, Marisa A. Jeffries, Akshata Almad, Asako Takanohashi, Quasar Saleem Padiath, Judith B. Grinspan, Eric D. Marsh, 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.
Molecular Therapy · Jan 20, 2026Peer-reviewedNeuroscience