Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy

Molecular Therapy 2026 Open access

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Authors: Sunetra Sase, Julia L. Hacker, Prabhat R. Napit, Anjali Bhagavatula, Sarah Woidill, Annemarie D’Alessandro, Marisa A. Jeffries, Akshata Almad, Asako Takanohashi, Quasar S. Padiath, Judith B. Grinspan, Eric D. Marsh
+1 more Adeline Vanderver
DOI: 10.1016/j.ymthe.2026.01.016
Cited by: 1
References: 56

Fields of Study & Subjects

RNA regulation and disease Nuclear Structure and Function Neurogenesis and neuroplasticity mechanisms

Publication details

Journal / source
Molecular Therapy
Publisher
Elsevier BV
Volume
34
Issue
5
Pages
2923–2943
Publication date
20 January 2026
Language
EN