Segmental duplications involving the alpha-globin gene cluster are causing beta-thalassemia intermedia phenotypes in beta-thalassemia heterozygous patients. 2008

C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
Hemoglobinopathies Laboratory, Leiden University Medical Center, Leiden, The Netherlands.

We describe two cases of simple heterozygosity for the common beta degrees -thalassemia mutation beta 39 (C-->T), both presenting with a thalassemia intermedia phenotype. In both cases synergic effect deriving from membrane defects or red cell enzyme deficiencies were excluded. In one case a triplication of the alpha-globin genes was found which did not justify the severity of the transfusion-dependent phenotype. Multiplex ligation-dependent probe amplification (MLPA) analysis of the alpha-globin gene cluster revealed two new rearrangements, consisting of a full duplication of the alpha-globin genes locus including the upstream regulatory element. In one case the duplication was in the presence of the common anti-alpha(3.7) triplication in trans, resulting in a total of 7 active alpha-globin genes. In the other case the duplicated allele and the normal allele in trans resulted into a total of 6 active alpha-globin genes. We report the clinical and hematological data and the molecular analysis and discuss the occurrence of alpha-globin genes duplication defects in cases of beta-thalassemia heterozygotes with thalassemia intermedia phenotypes.

UI MeSH Term Description Entries
D008875 Middle Aged An adult aged 45 - 64 years. Middle Age
D009154 Mutation Any detectable and heritable change in the genetic material that causes a change in the GENOTYPE and which is transmitted to daughter cells and to succeeding generations. Mutations
D010641 Phenotype The outward appearance of the individual. It is the product of interactions between genes, and between the GENOTYPE and the environment. Phenotypes
D011110 Polymorphism, Genetic The regular and simultaneous occurrence in a single interbreeding population of two or more discontinuous genotypes. The concept includes differences in genotypes ranging in size from a single nucleotide site (POLYMORPHISM, SINGLE NUCLEOTIDE) to large nucleotide sequences visible at a chromosomal level. Gene Polymorphism,Genetic Polymorphism,Polymorphism (Genetics),Genetic Polymorphisms,Gene Polymorphisms,Polymorphism, Gene,Polymorphisms (Genetics),Polymorphisms, Gene,Polymorphisms, Genetic
D005260 Female Females
D005810 Multigene Family A set of genes descended by duplication and variation from some ancestral gene. Such genes may be clustered together on the same chromosome or dispersed on different chromosomes. Examples of multigene families include those that encode the hemoglobins, immunoglobulins, histocompatibility antigens, actins, tubulins, keratins, collagens, heat shock proteins, salivary glue proteins, chorion proteins, cuticle proteins, yolk proteins, and phaseolins, as well as histones, ribosomal RNA, and transfer RNA genes. The latter three are examples of reiterated genes, where hundreds of identical genes are present in a tandem array. (King & Stanfield, A Dictionary of Genetics, 4th ed) Gene Clusters,Genes, Reiterated,Cluster, Gene,Clusters, Gene,Families, Multigene,Family, Multigene,Gene Cluster,Gene, Reiterated,Multigene Families,Reiterated Gene,Reiterated Genes
D005838 Genotype The genetic constitution of the individual, comprising the ALLELES present at each GENETIC LOCUS. Genogroup,Genogroups,Genotypes
D005914 Globins A superfamily of proteins containing the globin fold which is composed of 6-8 alpha helices arranged in a characterstic HEME enclosing structure. Globin
D006579 Heterozygote An individual having different alleles at one or more loci regarding a specific character. Carriers, Genetic,Genetic Carriers,Carrier, Genetic,Genetic Carrier,Heterozygotes
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man

Related Publications

C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
March 2010, Blood cells, molecules & diseases,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
January 1985, Acta haematologica,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
January 1993, Annals of hematology,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
January 1983, Human genetics,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
October 2009, Haematologica,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
December 1991, American journal of hematology,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
January 1998, American journal of hematology,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
May 1987, British journal of haematology,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
May 2024, American journal of hematology,
C L Harteveld, and C Refaldi, and E Cassinerio, and M D Cappellini, and P C Giordano
October 1988, American journal of hematology,
Copied contents to your clipboard!