Target intelligence / Profile preview

Transmembrane Domain 1 of CFTR (TMD1)

Target
TMD1
Molecular classification
Transmembrane domain, Protein domain, ABC transporter
01

Overview

Transmembrane Domain 1 (TMD1) of the CFTR protein is a critical structural and functional component of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), an ATP-binding cassette (ABC) transporter-class ion channel. TMD1, together with TMD2, forms the core transmembrane pore that allows selective passage of anions such as chloride (Cl-) and bicarbonate (HCO3-). Mutations affecting residues within or near TMD1 can disrupt proper folding, trafficking to the plasma membrane, or function of CFTR—leading to cystic fibrosis phenotypes depending on mutation severity.

Other names
CFTR TMD1TMD1
02

Mechanism of action

CFTR modulators (e.g., correctors, potentiators) affect the folding, trafficking, or gating of the CFTR protein, including its transmembrane domains.

03

Biological functions

Ion channel pore formationChloride ion transportBicarbonate ion transportChannel gating
04

Disease associations

Cystic fibrosis
05

Safety considerations

Off-target effects of CFTR modulatorsVariability in response to CFTR modulators based on mutation type.

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