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Hum Mutat ; 13(2): 146-53, 1999.
Artigo em Inglês | MEDLINE | ID: mdl-10094551

RESUMO

Denys-Drash and Frasier syndromes are rare human disorders that associate nephropathy with gonadal and genital abnormalities. In DDS there is a predisposition to Wilms' tumor. Heterozygous point mutations in the Wilms' tumor, type1 gene (WT1), particularly those altering the zinc finger (ZF) encoding exons, have been reported in most DDS patients, while mutations in intron 9 of the same gene cause FS. This paper describes two cases of DDS, one FS and one patient with Wilm's tumor and intersex genitalia, in which mutations were searched by sequencing the exons 8 and 9 of WT1 gene. Patient 1 carried a missense point mutation in exon 8 (ZF2), converting a CGA-Arg codon to a TGA-stop codon. Patient 2 presented a single nucleotide deletion within exon 9 (ZF3) introducing a premature chain termination at codon 398. Patients 3 and 4 had a C-->T transition at position +4 of the second alternative splice donor site of exon 9 (this mutation was detected in peripheral blood and in tumor derived DNA of patient 3). However, patient 3 had previously developed a Wilms' tumor. This is the first case of Wilms' tumor development in a phenotypically and genetically confirmed case of FS.


Assuntos
Genes do Tumor de Wilms/genética , Disgenesia Gonadal/genética , Falência Renal Crônica/genética , Mutação/genética , Splicing de RNA/genética , Anormalidades Urogenitais/genética , Tumor de Wilms/genética , Adolescente , Sequência de Bases , Criança , Análise Mutacional de DNA , Feminino , Humanos , Masculino , Dados de Sequência Molecular , Síndrome , Tumor de Wilms/complicações
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