Restoring the Balance: CRISPRa-Driven β-Tubulin Compensation as a Strategy for Tubulinopathy Treatment
By increasing the expression of various beta-tubulin proteins, this work demonstrates a restoration of the microtubule network and primary cilia formation and develops a mutation-independent treatment strategy based on the upregulation of non-mutated beta-tubulin isotype protein using CRISPR-Cas9 activation.
Sydney Steiman, Ciara Kinsella, Yi-Xiang Zhang et al.
· International Journal of Mol... · 0 citations