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Ciliopathies: Does HDAC6 Represent a New Therapeutic Target?

Journal

TRENDS IN PHARMACOLOGICAL SCIENCES
Volume 37, Issue 2, Pages 114-119

Publisher

ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tips.2015.11.002

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Funding

  1. National Basic Research Program of China [2012CB945002]

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Cilia are cellular appendages with critical roles in sensing and transducing environmental signals and guiding fluid flow. Consistent with these diverse activities, defects in ciliary structure or function have been implicated in a variety of human diseases, collectively known as 'ciliopathies'. Histone deacetylase 6 (HDAC6) is a unique cytoplasmic enzyme that regulates many biological processes through its deacetylase and ubiquitin-binding activities. There is accumulating evidence that HDAC6 is a major driver of ciliary disassembly. Small-molecule compounds that inhibit HDAC6 have been demonstrated to restore ciliary structure and function in several different ciliopathies. Here, we discuss recent findings that highlight the important role for HDAC6 in mediating ciliary disassembly and the potential for HDAC6-selective inhibitors as therapeutics for specific ciliopathies.

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