Skip to main content
U.S. Department of Energy
Office of Scientific and Technical Information

A novel donor splice site in intron 11 of the CFTR gene, created by mutation 1811 + 1.6kbA {yields} G, produces a new exon: High frequency in spanish cystic fibrosis chromosomes and association with severe phenotype

Journal Article · · American Journal of Human Genetics
OSTI ID:70439
; ; ; ; ;  [1]; ;  [2];  [3]
  1. Cancer Research Institute, Barcelona (Spain)
  2. Medizinische Hochschule Hannover (Germany)
  3. Institut Cochin de Genetique Moleculaire, Paris (France)

mRNA analysis of the cystic fibrosis transmembrane regulator (CFTR) gene in tissues of cystic fibrosis (CF) patients has allowed us to detect a cryptic exon. The new exon involves 49 base pairs between exons 11 and 12 and is due to a point mutation (1811+1.6bA{yields}G) that creates a new donor splice site in intron 11. Semiquantitative mRNA analysis showed that 1811+1.6kbA{r_arrow}G-mRNA was 5-10-fold less abundant than {triangle}F508 mRNA. Mutations 1811+1.6kbA{yields}G was found in 21 Spanish and 1 German CF chromosome(s), making it the fourth-most-frequent mutation (2%) in the Spanish population. Individuals with genotype {triangle}F508/1811+1.6kbA{yields}G have only 1%-3% of normal CFTR mRNA. This loss of 97% of normal CFTR mRNA must be responsible for the pancreatic insufficiency and for the severe CF phenotype in these patients. 30 refs., 3 figs., 2 tabs.

OSTI ID:
70439
Journal Information:
American Journal of Human Genetics, Journal Name: American Journal of Human Genetics Journal Issue: 3 Vol. 56; ISSN AJHGAG; ISSN 0002-9297
Country of Publication:
United States
Language:
English