A new exon created by intronic insertion of a rearranged LINE-1 element as the cause of chronic granulomatous disease

Eur J Hum Genet. 2000 Sep;8(9):697-703. doi: 10.1038/sj.ejhg.5200523.

Abstract

Long interspersed nuclear element-1 (LINE-1) or L1 elements are DNA elements present in the genome in high copy number and capable of active retrotransposition. Here we present a patient with severe chronic granulomatous disease (CGD) caused by insertion of an L1 sequence into intron 5 of the X-lined gene CYBB. Due to internal rearrangements, the insert introduced new splice sites into the intron. This resulted in a highly heterogeneous splicing pattern with introduction of two L1 fragments as new exons into the transcripts and concomitant skipping of exonic coding sequence. Because no wild-type cDNA was found, this mechanism is probably responsible for the patient's phenotype. The L1 fragment, which belongs to the Ta subset of transcriptionally active LINEs, illustrates a new mechanism by which these elements can modify the transcribed coding sequence of genes.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Alternative Splicing / genetics
  • Base Sequence
  • Child, Preschool
  • Exons / genetics*
  • Fatal Outcome
  • Granulomatous Disease, Chronic / enzymology
  • Granulomatous Disease, Chronic / etiology
  • Granulomatous Disease, Chronic / genetics*
  • Humans
  • Infant, Newborn
  • Introns / genetics*
  • Long Interspersed Nucleotide Elements / genetics*
  • Male
  • Membrane Glycoproteins / genetics
  • Molecular Sequence Data
  • Mutagenesis, Insertional / genetics*
  • NADPH Oxidase 2
  • NADPH Oxidases / deficiency
  • NADPH Oxidases / genetics
  • Recombination, Genetic*

Substances

  • Membrane Glycoproteins
  • CYBB protein, human
  • NADPH Oxidase 2
  • NADPH Oxidases