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Target intelligence / Profile preview
Cystic Fibrosis Transmembrane Conductance Regulator Nucleotide-Binding Domain 1 (CFTR NBD1)
- Target
- CFTR NBD1
- Molecular classification
- ABC transporter domain, Nucleotide-binding domain
01
Overview
The cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel. The nucleotide-binding domain 1 (NBD1) is one of two cytosolic nucleotide-binding domains within CFTR that are essential for its function as an ATP-gated ion channel. Mutations affecting structure or function of CFTR, particularly those impacting folding or stability of NBD1 such as ΔF508, lead to cystic fibrosis due to defective chloride transport.
- Other names
- NBD1CFTR NBD-1CFTR Nucleotide-Binding Domain 1
02
Mechanism of action
Modulation of NBD1 folding and stability to enhance CFTR function.
03
Biological functions
ATP bindingATP hydrolysisRegulation of CFTR channel gatingProtein foldingProtein stability
04
Disease associations
Cystic fibrosis
05
Safety considerations
Off-target effects on other ABC transportersDevelopment of resistance mutations
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