title: Immunochemical analysis of mutant CFTR in lung explants.
abstract: OBJECTIVE
  Knowledge about the abundance and distribution of CFTR protein glycoforms in native lung tissue is scarce. For upcoming studies with correctors and potentiators for CFTR it is important to get more information about mutant CFTR protein biochemistry. Target for novel treatment is the most afflicted organ in cystic fibrosis (CF), the lung.
  
  
  METHODS
  Lung tissue sampled from patients with CF and non-CF donors prior to lung transplantation was examined for CFTR-immunoreactive signals by immunoblot. Quantitation of the immunoreactive signals was carried out by densitometry.
  
  
  RESULTS
  The complex-glycosylated and mannose-rich CFTR isoforms were present in all non-CF specimens, whereas no or only the immature CFTR isoform was visible in CF samples. Whereas some complex-glycosylated CFTR was often present in rectal biopsies of F508del homozygous subjects, no mature CFTR was detectable in CF lungs at the stage of terminal respiratory insufficiency.
  
  
  CONCLUSIONS
  Immunoblot analysis of CFTR in lung tissue is feasible, but in context of the upcoming studies of CFTR correctors and potentiators rectal biopsies seem to be a more appropriate choice because of their safe and repeatable excision.
authors: Klinische Forschergruppe, OE 6710, Medizinische Hochschule Hannover, Hannover, Germany. barneveld.andrea.van@mh-hannover.de
authors: Andrea
authors: A
authors: van Barneveld
authors: Isabell
authors: I
authors: Zander
authors: Rebecca
authors: R
authors: Hyde
authors: Florian
authors: F
authors: Länger
authors: André
authors: A
authors: Simon
authors: Marcus
authors: M
authors: Krüger
authors: Manfred
authors: M
authors: Ballmann
authors: Nico
authors: N
authors: Derichs
authors: Burkhard
authors: B
authors: Tümmler
mesh_terms: D003550:Cystic Fibrosis / Q000276:immunology / Q000378:metabolism / Q000473:pathology; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000378:metabolism*; D006031:Glycosylation; D006720:Homozygote; D006801:Humans; D008168:Lung / Q000378:metabolism* / Q000473:pathology; D016040:Lung Transplantation; D009154:Mutation; D020033:Protein Isoforms / Q000235:genetics / Q000378:metabolism
chemical_list: D020033:Protein Isoforms; D019005:Cystic Fibrosis Transmembrane Conductance Regulator