High frequency of ribosomal protein gene deletions in Italian Diamond-Blackfan anemia patients detected by multiplex ligation-dependent probe amplification assay
High frequency of ribosomal protein gene deletions in Italian Diamond-Blackfan anemia patients detected by multiplex ligation-dependent probe amplification assay
Diamond-Blackfan anemia is an autosomal dominant disease due to mutations in nine ribosomal protein encoding genes. Because most mutations are loss of function and detected by direct sequencing of coding exons, we reasoned that part of the approximately 50% mutation negative patients may have carried a copy number variant of ribosomal protein genes. As a proof of concept, we designed a multiplex ligation-dependent probe amplification assay targeted to screen the six genes that are most frequently mutated in Diamond-Blackfan anemia patients: RPS17, RPS19, RPS26, RPL5, RPL11, and RPL35A. Using this assay we showed that deletions represent approximately 20% of all mutations. The combination of sequencing and multiplex ligation-dependent probe amplification analysis of these six genes allows the genetic characterization of approximately 65% of patients, showing that Diamond-Blackfan anemia is indisputably a ribosomopathy.
- Istituti di Ricovero e Cura a Carattere Scientifico Italy
- Boston Children's Hospital United States
- Ospedale Regina Margherita Italy
- University of Turin Italy
- Bambino Gesù Children's Hospital Italy
Ribosomal Proteins, Diamond-Blackfan anemia; multiplex ligation-dependent probe amplification; ribosomal protein gene deletion, Prognosis, Gene Duplication, 616, Mutation, Humans, Diseases of the blood and blood-forming organs, Registries, RC633-647.5, Multiplex Polymerase Chain Reaction, Gene Deletion, Anemia, Diamond-Blackfan
Ribosomal Proteins, Diamond-Blackfan anemia; multiplex ligation-dependent probe amplification; ribosomal protein gene deletion, Prognosis, Gene Duplication, 616, Mutation, Humans, Diseases of the blood and blood-forming organs, Registries, RC633-647.5, Multiplex Polymerase Chain Reaction, Gene Deletion, Anemia, Diamond-Blackfan
13 Research products, page 1 of 2
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
chevron_left - 1
- 2
chevron_right
citations This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).47 popularity This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.Top 10% influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).Top 10% impulse This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.Top 10%
