Mutation analysis in nephronophthisis using a combined approach of homozygosity mapping, CEL I endonuclease cleavage, and direct sequencing

Hum Mutat. 2008 Mar;29(3):418-26. doi: 10.1002/humu.20669.

Abstract

Nephronophthisis (NPHP), an autosomal recessive kidney disease, is the most frequent genetic cause of chronic renal failure in the first three decades of life. Mutations in eight genes (NPHP1-8) have been identified. We here describe a combined approach for mutation screening of NPHP1, NPHP2, NPHP3, NPHP4, and NPHP5 in a worldwide cohort of 470 unrelated patients with NPHP. First, homozygous NPHP1 deletions were detected in 97 patients (21%) by multiplex PCR. Second, 25 patients with infantile NPHP were screened for mutations in inversin (NPHP2/INVS). We detected a novel compound heterozygous frameshift mutation (p.[Q485fs]+[R687fs]), and a homozygous nonsense mutation (p.R899X). Third, 37 patients presenting with NPHP and retinitis pigmentosa (Senior-Løken syndrome [SLS]) were screened for NPHP5/IQCB1 mutations by direct sequencing. We discovered five different (three novel) homozygous premature termination codon (PTC) mutations (p.F142fsX; p.R461X; p.R489X; p.W444X; and c.488-1G>A). The remaining 366 patients were further investigated for mutations in NPHP1, NPHP3, and NPHP4. We applied a "homozygosity only" strategy and typed three highly polymorphic microsatellite markers at the respective loci. A total of 32, eight, and 14 patients showed homozygosity, and were screened by heteroduplex crude celery extract (CEL I) endonuclease digests. The sensitivity of CEL I was established as 92%, as it detected 73 out of 79 different known mutations simply on agarose gels. A total of 10 novel PTC mutations were found in NPHP1 (p.P186fs, p.R347X, p.V492fs, p.Y509X, and c.1884+1G>A), in NPHP3 (c.3812+2T>C and p.R1259X), and in NPHP4 (p.R59X, p.T1004fs, and p.V1091fs). The combined homozygosity mapping and CEL I endonuclease mutation analysis approach allowed us to identify rare mutations in a large cohort of patients at low cost.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adaptor Proteins, Signal Transducing
  • Antigens, Neoplasm
  • Base Sequence
  • Calmodulin-Binding Proteins / genetics
  • Cell Cycle Proteins
  • Chromosome Mapping
  • Cytoskeletal Proteins
  • DNA Mutational Analysis
  • DNA Primers / genetics
  • Endonucleases
  • Female
  • Gene Deletion
  • Genes, Recessive
  • Homozygote
  • Humans
  • Kidney Diseases / complications
  • Kidney Diseases / genetics*
  • Male
  • Membrane Proteins
  • Microsatellite Repeats
  • Mutation*
  • Neoplasm Proteins
  • Point Mutation
  • Proteins / genetics*
  • Retinitis Pigmentosa / complications
  • Retinitis Pigmentosa / genetics
  • Reverse Transcriptase Polymerase Chain Reaction
  • Transcription Factors / genetics

Substances

  • Adaptor Proteins, Signal Transducing
  • Antigens, Neoplasm
  • Calmodulin-Binding Proteins
  • Cell Cycle Proteins
  • Cep290 protein, human
  • Cytoskeletal Proteins
  • DNA Primers
  • INVS protein, human
  • IQCB1 protein, human
  • Membrane Proteins
  • NPHP1 protein, human
  • NPHP4 protein, human
  • Neoplasm Proteins
  • Proteins
  • Transcription Factors
  • Endonucleases
  • CEL I nuclease