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CFTR

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation

106 views | Jan 18 2019

Accurso FJ et al. evaluated the safety and adverse-event profile of VX-770 showed that VX-770 was associated with within-subject improvements in CFTR and lung function. These findings provide support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis. [Read the Full Post]

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

120 views | Jan 17 2019

Rowe SM et al. showed that CFTR modulator therapy with tezacaftor-ivacaftor or ivacaftor alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual-function mutation. [Read the Full Post]

CFTR protects against vascular inflammation and atherogenesis in apolipoprotein E-deficient mice

98 views | Jan 08 2019

Li Z et al. suggested that CFTR may present a potential therapeutic target for the treatment of vascular inflammation and development of atherosclerotic disease. [Read the Full Post]

High-expressing cystic fibrosis transmembrane conductance regulator interacts with histone deacetylase 2 to promote the development of Ph+ leukemia through the HDAC2-mediated PTEN pathway

0 views | Oct 04 2018

Yan T et al. provides strong evidence that high-expression CFTR plays an important role in the development of Ph+ leukemia through the HDAC2-mediated PTEN pathway. [Read the Full Post]

Tissue-specific variation in nonsense mutant transcript level and drug-induced read-through efficiency in the Cln1(R151X) mouse model of INC

508 views | Nov 22 2017

Thada V et al. identified a new challenge/hurdle for read-through drug therapy: variable efficiency of read-through therapy in the different tissues/organs because of tissue-specific variations in nonsense mutant transcript levels. [Read the Full Post]

Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds

0 views | Oct 15 2017

Langron E et al. showed that most known potentiators have a negative influence on F508del-CFTR biogenesis/stability, which means membrane exposure needs to be monitored early during the development of drugs targeting CFTR. The combined use of the two fluorescence assays described here provides a useful tool for the identification of improved potentiators and correctors. The assays could also prove useful for basic scientific investigations on F508del-CFTR, and other CF-causing mutations. [Read the Full Post]

Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis

578 views | Oct 14 2017

Bali V et al. emphasized that stabilization of ΔF508 CFTR band B in the ER might improve its functional rescue by Orkambi. [Read the Full Post]

Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds

757 views | Oct 04 2017

Langron E et al. showed that most known potentiators have a negative influence on F508del-CFTR biogenesis/stability, which means membrane exposure needs to be monitored early during the development of drugs targeting CFTR. The combined use of the two fluorescence assays described here provides a useful tool for the identification of improved potentiators and correctors. The assays could also prove useful for basic scientific investigations on F508del-CFTR, and other CF-causing mutations. [Read the Full Post]

Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis

1102 views | Nov 14 2016

Bali V et al. found that stabilization of ΔF508 CFTR band B in the ER might improve its functional rescue by Orkambi. [Read the Full Post]

VX 770 is a drug approved for patients with a certain mutation of cystic fibrosis

2304 views | Dec 09 2013

Ivacaftor (VX-770, Kalydeco) is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively. [Read the Full Post]