ID

45460

Description

Principal Investigator: Evan E. Eichler, PhD, Department of Genome Sciences, University of Washington School of Medicine, Seattle, Washington, USA; Howard Hughes Medical Institute, Seattle, WA, USA MeSH: Autistic Disorder,Child Development Disorders, Pervasive https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000482 It is well established that autism spectrum disorders (ASD) have a strong genetic component; however, for at least 70% of cases, the underlying genetic cause is unknown. Under the hypothesis that de novo mutations underlie a substantial fraction of the risk for developing ASD in families with no previous history of ASD or related phenotypes-so-called sporadic or simplex families-we sequenced all coding regions of the genome (the exome) for parent-child trios exhibiting sporadic ASD, including 189 new trios and 20 that were previously reported. Additionally, we also sequenced the exomes of 50 unaffected siblings corresponding to these new (n = 31) and previously reported trios (n = 19), for a total of 617 individual exomes from 209 families deposited in dbGaP. Here we show that de novo point mutations are overwhelmingly paternal in origin (4:1 bias) and positively correlated with paternal age, consistent with the modest increased risk for children of older fathers to develop ASD. Moreover, 39% (49 of 126) of the most severe or disruptive de novo mutations map to a highly interconnected beta-catenin/chromatin remodelling protein network ranked significantly for autism candidate genes. In proband exomes, recurrent protein-altering mutations were observed in two genes: CHD8 and NTNG1. Mutation screening of six candidate genes in 1,703 ASD probands identified additional de novo, protein-altering mutations in GRIN2B, LAMC3 and SCN1A. Combined with copy number variant (CNV) data, these results indicate extreme locus heterogeneity but also provide a target for future discovery, diagnostics and therapeutics.

Link

dbGap-study=phs000482

Keywords

  1. 10/20/22 10/20/22 - Simon Heim
  2. 12/12/22 12/12/22 - Kristina Keller
Copyright Holder

Evan E. Eichler, PhD, Department of Genome Sciences, University of Washington School of Medicine, Seattle, Washington, USA; Howard Hughes Medical Institute, Seattle, WA, USA

Uploaded on

December 12, 2022

DOI

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License

Creative Commons BY 4.0

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dbGaP phs000482 Sporadic Autism Exomes Reveal a Highly Interconnected Protein Network of De Novo Mutations

Eligibility Criteria

Inclusion and exclusion criteria
Description

Inclusion and exclusion criteria

Trios (Father, Mother, and Proband) (n=189)
Description

Elig.phs000482.v1.p1.1

Data type

boolean

Alias
UMLS CUI [1,1]
C0026591
UMLS CUI [1,2]
C0015671
UMLS CUI [1,3]
C1948021
Exclusions:
Description

Elig.phs000482.v1.p1.2

Data type

boolean

Alias
UMLS CUI [1,1]
C0680251
Known large CNVs as identified in Sanders et al., 2011. [PMID: 21658581]
Description

Elig.phs000482.v1.p1.3

Data type

boolean

Alias
UMLS CUI [1,1]
C0680251
UMLS CUI [1,2]
C1511518
Included:
Description

Elig.phs000482.v1.p1.4

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
86 males affected with ASD but not intellectual disability
Description

Elig.phs000482.v1.p1.5

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0086582
UMLS CUI [1,3]
C1510586
UMLS CUI [1,4]
C1298908
UMLS CUI [1,5]
C3714756
47 males affected with ASD and intellectual disability
Description

Elig.phs000482.v1.p1.6

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0086582
UMLS CUI [1,3]
C1510586
UMLS CUI [1,4]
C3714756
30 females affected with ASD but not intellectual disability
Description

Elig.phs000482.v1.p1.7

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0086287
UMLS CUI [1,3]
C1510586
UMLS CUI [1,4]
C1298908
UMLS CUI [1,5]
C3714756
26 females with ASD and intellectual disability
Description

Elig.phs000482.v1.p1.8

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0086287
UMLS CUI [1,3]
C1510586
UMLS CUI [1,4]
C3714756
Unaffected Siblings (n=50)
Description

Elig.phs000482.v1.p1.9

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C2986417
UMLS CUI [1,3]
C0037047
Exclusions:
Description

Elig.phs000482.v1.p1.10

Data type

boolean

Alias
UMLS CUI [1,1]
C0680251
None
Description

Elig.phs000482.v1.p1.11

Data type

boolean

Alias
UMLS CUI [1,1]
C0680251
UMLS CUI [1,2]
C0549184
Included:
Description

Elig.phs000482.v1.p1.12

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
19 siblings matching probands from O'Roak et al., 2011. [PMID: 21572417]
Description

Elig.phs000482.v1.p1.13

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0037047
UMLS CUI [1,3]
C0150103
UMLS CUI [1,4]
C1948021
31 siblings matching the probands listed above.
Description

Elig.phs000482.v1.p1.14

Data type

boolean

Alias
UMLS CUI [1,1]
C1512693
UMLS CUI [1,2]
C0037047
UMLS CUI [1,3]
C0150103
UMLS CUI [1,4]
C1948021

Similar models

Eligibility Criteria

Name
Type
Description | Question | Decode (Coded Value)
Data type
Alias
Item Group
Inclusion and exclusion criteria
Elig.phs000482.v1.p1.1
Item
Trios (Father, Mother, and Proband) (n=189)
boolean
C0026591 (UMLS CUI [1,1])
C0015671 (UMLS CUI [1,2])
C1948021 (UMLS CUI [1,3])
Elig.phs000482.v1.p1.2
Item
Exclusions:
boolean
C0680251 (UMLS CUI [1,1])
Elig.phs000482.v1.p1.3
Item
Known large CNVs as identified in Sanders et al., 2011. [PMID: 21658581]
boolean
C0680251 (UMLS CUI [1,1])
C1511518 (UMLS CUI [1,2])
Elig.phs000482.v1.p1.4
Item
Included:
boolean
C1512693 (UMLS CUI [1,1])
Elig.phs000482.v1.p1.5
Item
86 males affected with ASD but not intellectual disability
boolean
C1512693 (UMLS CUI [1,1])
C0086582 (UMLS CUI [1,2])
C1510586 (UMLS CUI [1,3])
C1298908 (UMLS CUI [1,4])
C3714756 (UMLS CUI [1,5])
Elig.phs000482.v1.p1.6
Item
47 males affected with ASD and intellectual disability
boolean
C1512693 (UMLS CUI [1,1])
C0086582 (UMLS CUI [1,2])
C1510586 (UMLS CUI [1,3])
C3714756 (UMLS CUI [1,4])
Elig.phs000482.v1.p1.7
Item
30 females affected with ASD but not intellectual disability
boolean
C1512693 (UMLS CUI [1,1])
C0086287 (UMLS CUI [1,2])
C1510586 (UMLS CUI [1,3])
C1298908 (UMLS CUI [1,4])
C3714756 (UMLS CUI [1,5])
Elig.phs000482.v1.p1.8
Item
26 females with ASD and intellectual disability
boolean
C1512693 (UMLS CUI [1,1])
C0086287 (UMLS CUI [1,2])
C1510586 (UMLS CUI [1,3])
C3714756 (UMLS CUI [1,4])
Elig.phs000482.v1.p1.9
Item
Unaffected Siblings (n=50)
boolean
C1512693 (UMLS CUI [1,1])
C2986417 (UMLS CUI [1,2])
C0037047 (UMLS CUI [1,3])
Elig.phs000482.v1.p1.10
Item
Exclusions:
boolean
C0680251 (UMLS CUI [1,1])
Elig.phs000482.v1.p1.11
Item
None
boolean
C0680251 (UMLS CUI [1,1])
C0549184 (UMLS CUI [1,2])
Elig.phs000482.v1.p1.12
Item
Included:
boolean
C1512693 (UMLS CUI [1,1])
Elig.phs000482.v1.p1.13
Item
19 siblings matching probands from O'Roak et al., 2011. [PMID: 21572417]
boolean
C1512693 (UMLS CUI [1,1])
C0037047 (UMLS CUI [1,2])
C0150103 (UMLS CUI [1,3])
C1948021 (UMLS CUI [1,4])
Elig.phs000482.v1.p1.14
Item
31 siblings matching the probands listed above.
boolean
C1512693 (UMLS CUI [1,1])
C0037047 (UMLS CUI [1,2])
C0150103 (UMLS CUI [1,3])
C1948021 (UMLS CUI [1,4])

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