Article

HDAC Inhibitors Rescue Multiple Disease-Causing CFTR Variants

2018-08-24

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 respond of the disease population to any therapeutic. An ever-growing body of evidence has shown that histone deacetylase (HDAC) inhibitors (HDACi) can have significant benefit in correcting pr...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 28d0c45e-99d6-5736-b650-641d86d3ff99
DOI: 10.1101/399451

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

HDAC Inhibitors Rescue Multiple Disease-Causing CFTR VariantsDOI 10.1101/399451Read

HDAC inhibitors rescue multiple disease-causing CFTR variantsSemantic proximity Read

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis.Semantic proximity Read

Integrative genomic meta-analysis reveals novel molecular insights into cystic fibrosis and ΔF508-CFTR rescue.Semantic proximity Read

Chasing a Breath of Fresh Air in Cystic Fibrosis (CF): Therapeutic Potential of Selective HDAC6 Inhibitors to Tackle Multiple Pathways in CF PathophysiologySemantic proximity Read

Distinct proteostasis states drive pharmacologic chaperone susceptibility for Cystic Fibrosis Transmembrane Conductance Regulator misfolding mutantsSemantic proximity Read

Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding statesSemantic proximity Read

Distinct proteostasis states drive pharmacologic chaperone susceptibility for cystic fibrosis transmembrane conductance regulator misfolding mutants.Semantic proximity Read

Proteostasis Landscapes of Cystic Fibrosis Variants Reveals Drug Response VulnerabilitySemantic proximity Read

CFTR Folding: From Structure and Proteostasis to Cystic Fibrosis Personalized Medicine.Semantic proximity Read

The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CFSemantic proximity Read

Inhibition of the QPCT-PDIA4 axis rescues ΔF508 and N1303K CFTR in cystic fibrosis.Semantic proximity Read

Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states.Semantic proximity Read