Rescue of functional DeltaF508-CFTR channels by co-expression with truncated CFTR constructs in COS-1 cells

FEBS Lett. 2003 Nov 6;554(1-2):173-8. doi: 10.1016/s0014-5793(03)01162-1.

Abstract

The most frequent mutant variant of the cystic fibrosis transmembrane conductance regulator (CFTR), DeltaF508-CFTR, is misprocessed and subsequently degraded in the endoplasmic reticulum. Using the patch-clamp technique, we showed that co-expressions of DeltaF508-CFTR with the N-terminal CFTR truncates containing bi-arginine (RXR) retention/retrieval motifs result in a functional rescue of the DeltaF508-CFTR mutant channel in COS-1 cells. This DeltaF508-CFTR rescue process was strongly impaired when truncated CFTR constructs possessed either the DeltaF508 mutation or arginine-to-lysine mutations in RXRs. In conclusions, our data demonstrated that expression of truncated CFTR constructs could be a novel promising approach to improve maturation of DeltaF508-CFTR channels.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • COS Cells
  • Chlorides / metabolism
  • Cyclic AMP / pharmacology
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • Cystic Fibrosis Transmembrane Conductance Regulator / physiology
  • Electrophysiology
  • Endoplasmic Reticulum / metabolism
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Mutation
  • Patch-Clamp Techniques
  • Protein Engineering
  • Transfection

Substances

  • Chlorides
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Cyclic AMP