ID

45307

Beschrijving

Principal Investigator: Hanna Gazda, Children's Hospital Boston, Boston, MA, USA MeSH: Anemia, Dyserythropoietic, Congenital,Anemia, Diamond-Blackfan https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000474 In this study we have performed exome sequencing using the hybrid capture method that has previously been described (Gnirke et al., Nature Biotechnology, 2009) on genomic DNA samples from patients with the congenital bone marrow failure syndrome that affects the erythroid lineage, specifically, Diamond-Blackfan anemia. This disease is characterized by a hypoplastic anemia and approximately 50% of cases are attributable to mutations in ribosomal protein gene subunits. The other 50% of cases do not have a known genetic etiology and the purpose of this study is to attempt to delineate such causes.

Link

dbGaP study = phs000474

Trefwoorden

  1. 19/10/22 19/10/22 - Adrian Schulz
Houder van rechten

Hanna Gazda, Children's Hospital Boston, Boston, MA, USA

Geüploaded op

19 de octubre de 2022

DOI

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Licentie

Creative Commons BY 4.0

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dbGaP phs000474 Biology and Molecular Analysis of Human Hematopoiesis Genetics

Eligibility Criteria

Inclusion and exclusion criteria
Beschrijving

Inclusion and exclusion criteria

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0680251
We have selected probands with Diamond-Blackfan anemia diagnosed based upon clinical and laboratory criteria and have excluded patients with known genetic mutations. Details of this cohort that was used in this study have been described in prior publications (Gazda et al., Am J Hum Genet, 2008). The third version of the study includes data for 217 additional subjects that have been used as a replication study.
Beschrijving

Elig.phs000474.v3.p2.1

Datatype

boolean

Alias
UMLS CUI [1,1]
C3846158

Similar models

Eligibility Criteria

Name
Type
Description | Question | Decode (Coded Value)
Datatype
Alias
Item Group
Inclusion and exclusion criteria
C1512693 (UMLS CUI [1,1])
C0680251 (UMLS CUI [1,2])
Elig.phs000474.v3.p2.1
Item
We have selected probands with Diamond-Blackfan anemia diagnosed based upon clinical and laboratory criteria and have excluded patients with known genetic mutations. Details of this cohort that was used in this study have been described in prior publications (Gazda et al., Am J Hum Genet, 2008). The third version of the study includes data for 217 additional subjects that have been used as a replication study.
boolean
C3846158 (UMLS CUI [1,1])

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