ID

46171

Beschreibung

Principal Investigator: Garry R. Cutting, MD, Johns Hopkins Medical Institutes, McKusick-Nathans Institute of Genetic Medicine, Baltimore, MD, USA MeSH: Cystic Fibrosis https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000556 The NHLBI "Grand Opportunity" Exome Sequencing Project (GO-ESP), a signature project of the NHLBI Recovery Act investment, was designed to identify genetic variants in coding regions (exons) of the human genome (the "exome") that are associated with heart, lung and blood diseases. These and related diseases that are of high impact to public health and individuals from diverse racial and ethnic groups will be studied. These data may help researchers understand the causes of disease, contributing to better ways to prevent, diagnose, and treat diseases, as well as determine whether to tailor prevention and treatments to specific populations. This could lead to more effective treatments and reduce the likelihood of side effects. GO-ESP is comprised of five collaborative components: 3 cohort consortia - HeartGO, LungGO, and WHISP - and 2 sequencing centers - BroadGO and SeattleGO. Cystic fibrosis (CF) patients without detectable disease-causing mutations in CFTR present a clinical phenotype very similar to, but possibly discrete from, classic CF. We hypothesize that the phenotype of these atypical CF patients is not due only to mutations in the carbonic anhydrase gene CA12 or in the genes encoding the epithelium sodium ion channel ENaC, but is due to mutations in novel genes that have yet to be associated with CF. The goal of this study is to determine the molecular etiology of this atypical CF.

Link

https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000556

Stichworte

  1. 02.11.22 02.11.22 - Simon Heim
  2. 29.01.25 29.01.25 - Akane Nishihara
Rechteinhaber

Garry R. Cutting, MD, Johns Hopkins Medical Institutes, McKusick-Nathans Institute of Genetic Medicine, Baltimore, MD, USA

Hochgeladen am

29. Januar 2025

DOI

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Lizenz

Creative Commons BY 4.0

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dbGaP phs000556 NHLBI GO-ESP: Family Studies (Atypical Cystic Fibrosis)

Eligibility Criteria

Inclusion and exclusion criteria
Beschreibung

Inclusion and exclusion criteria

Inclusion criteria are: elevated sweat chloride levels >40mM, pulmonary dysfunction (lung infections, reduced FEV1, or other respiratory symptoms), and lack of detectable disease-causing mutations in CFTR gene. Family members of individuals with these criteria are also included.
Beschreibung

Inclusion criteria are: elevated sweat chloride levels >40mM, pulmonary dysfunction (lung infections, reduced FEV1, or other respiratory symptoms), and lack of detectable disease-causing mutations in CFTR gene. Family members of individuals with these criteria are also included.

Datentyp

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C4746589
UMLS CUI [1,3]
C1709770
UMLS CUI [1,4]
C5208133
UMLS CUI [1,5]
C0086282
UMLS CUI [1,6]
C3897171
Exclusion criteria are: mutations in the CA12 gene or ENaC subunit genes which are predicted to be pathogenic.
Beschreibung

Exclusion criteria are: mutations in the CA12 gene or ENaC subunit genes which are predicted to be pathogenic.

Datentyp

boolean

Alias
UMLS CUI [1,1]
C0680251
UMLS CUI [1,2]
C1413041
UMLS CUI [1,3]
C0026882
UMLS CUI [1,4]
C0384156
UMLS CUI [1,5]
C1711351
UMLS CUI [1,6]
C0681842
UMLS CUI [1,7]
C3816499

Ähnliche Modelle

Eligibility Criteria

Name
Typ
Description | Question | Decode (Coded Value)
Datentyp
Alias
Item Group
Inclusion and exclusion criteria
Inclusion criteria are: elevated sweat chloride levels >40mM, pulmonary dysfunction (lung infections, reduced FEV1, or other respiratory symptoms), and lack of detectable disease-causing mutations in CFTR gene. Family members of individuals with these criteria are also included.
Item
Inclusion criteria are: elevated sweat chloride levels >40mM, pulmonary dysfunction (lung infections, reduced FEV1, or other respiratory symptoms), and lack of detectable disease-causing mutations in CFTR gene. Family members of individuals with these criteria are also included.
boolean
C1512693 (UMLS CUI [1,1])
C4746589 (UMLS CUI [1,2])
C1709770 (UMLS CUI [1,3])
C5208133 (UMLS CUI [1,4])
C0086282 (UMLS CUI [1,5])
C3897171 (UMLS CUI [1,6])
Exclusion criteria are: mutations in the CA12 gene or ENaC subunit genes which are predicted to be pathogenic.
Item
Exclusion criteria are: mutations in the CA12 gene or ENaC subunit genes which are predicted to be pathogenic.
boolean
C0680251 (UMLS CUI [1,1])
C1413041 (UMLS CUI [1,2])
C0026882 (UMLS CUI [1,3])
C0384156 (UMLS CUI [1,4])
C1711351 (UMLS CUI [1,5])
C0681842 (UMLS CUI [1,6])
C3816499 (UMLS CUI [1,7])

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