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Article

Restoring the Balance: CRISPRa-Driven β-Tubulin Compensation as a Strategy for Tubulinopathy Treatment.

International journal of molecular sciences - 3 Aug 2026

Steiman Sydney, Kinsella Ciara, Zhang Yixiang, Omer Safia, Brown Cadence, Forguson Georgiana, Elbakr Lujaina, Pastore Stephen, Ivakine Evgueni A

Abstract excerpt

Microtubules are essential cytoskeletal components comprising alpha- and beta-tubulin proteins that facilitate organelle positioning, cell migration, division, and intracellular trafficking. Mutations in tubulin genes can lead to tubulinopathies, a class of rare genetic neurodevelopmental disorders characterized by a range of brain malformations and other clinical features. Recent studies have shown that...

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Cilia
Ciliopathies
CRISPR-Cas Systems
Gene Editing
Microtubules

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PMID: 42589608
DOI: 10.3390/ijms27156954
PMCID: PMC13466901

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