Julia Liao Hacker

This study shows that deleting TUBB4A in human iPSCs reduces cell-autonomous deficits in oligodendrocytes and neurons associated with H-ABC, suggesting it could be a therapeutic strategy for TUBB4A-related leukodystrophy.

This study shows that suppressing Tubb4a with an ASO improves symptoms and extends lifespan in a mouse model of H-ABC leukodystrophy.

This paper develops a novel lentiviral vector for gene therapy of MLD that achieves high ARSA activity with minimal genomic integrations, improving safety and efficacy in preclinical models.