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Article

HDAC inhibitors rescue multiple disease-causing CFTR variants

5 Feb 2019

Abstract excerpt

Understanding the role of the epigenome in protein-misfolding diseases remains a challenge in light of genetic diversity found in the world-wide population revealed by human genome sequencing efforts and the highly variable response of the disease population to therapeutics. An ever-growing body of evidence has shown that histone deacetylase (HDAC) inhibitors (HDACi) can have significant benefit in correcting...

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PMID: 30753450
DOI: 10.1093/hmg/ddz026

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