Mutations in SBDS are associated with Shwachman-Diamond syndrome

Nat Genet. 2003 Jan;33(1):97-101. doi: 10.1038/ng1062. Epub 2002 Dec 23.

Abstract

Shwachman-Diamond syndrome (SDS; OMIM 260400) is an autosomal recessive disorder with clinical features that include pancreatic exocrine insufficiency, hematological dysfunction and skeletal abnormalities. Here, we report identification of disease-associated mutations in an uncharacterized gene, SBDS, in the interval of 1.9 cM at 7q11 previously shown to be associated with the disease. We report that SBDS has a 1.6-kb transcript and encodes a predicted protein of 250 amino acids. A pseudogene copy (SBDSP) with 97% nucleotide sequence identity resides in a locally duplicated genomic segment of 305 kb. We found recurring mutations resulting from gene conversion in 89% of unrelated individuals with SDS (141 of 158), with 60% (95 of 158) carrying two converted alleles. Converted segments consistently included at least one of two pseudogene-like sequence changes that result in protein truncation. SDBS is a member of a highly conserved protein family of unknown function with putative orthologs in diverse species including archaea and eukaryotes. Archaeal orthologs are located within highly conserved operons that include homologs of RNA-processing genes, suggesting that SDS may be caused by a deficiency in an aspect of RNA metabolism that is essential for development of the exocrine pancreas, hematopoiesis and chrondrogenesis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Base Sequence
  • Chromosomes, Human, Pair 7 / genetics
  • Conserved Sequence
  • DNA Mutational Analysis
  • Exocrine Pancreatic Insufficiency / genetics*
  • Female
  • Gene Conversion
  • Gene Expression Profiling
  • Hematologic Diseases / genetics*
  • Humans
  • Lod Score
  • Male
  • Molecular Sequence Data
  • Musculoskeletal Abnormalities / genetics*
  • Mutation
  • Proteins / genetics*
  • Pseudogenes / genetics
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Reverse Transcriptase Polymerase Chain Reaction
  • Syndrome

Substances

  • Proteins
  • RNA, Messenger
  • SBDS protein, human

Associated data

  • GENBANK/AC005236
  • GENBANK/AC006480
  • GENBANK/AC069291
  • GENBANK/AC073089
  • GENBANK/AC079920
  • GENBANK/AC091738
  • GENBANK/AC092536
  • GENBANK/AC105447
  • GENBANK/AC114731
  • GENBANK/AC114816
  • GENBANK/AK000961
  • GENBANK/AK001762
  • GENBANK/AK001779
  • GENBANK/AY169963
  • GENBANK/BJ013200
  • GENBANK/BJ025159
  • GENBANK/P70122
  • OMIM/260400
  • RefSeq/NP_013122
  • RefSeq/NP_126299
  • RefSeq/NP_147753
  • RefSeq/NP_247572
  • RefSeq/NP_280149
  • RefSeq/NP_342243
  • RefSeq/NP_394745
  • RefSeq/NP_497226
  • RefSeq/NP_559847
  • RefSeq/NP_597289
  • RefSeq/NP_613669
  • RefSeq/NP_616704
  • RefSeq/NP_648057