Cited by 706Genomics and Rare DiseasesPrenatal Screening and DiagnosticsMetabolism and Genetic Disorders

The DOI matches its Crossref record; 64 of the 66 references it cites were found in a registry or an index; 40 of the 80 sources its claims cite were checked for support.

Carrier Testing for Severe Childhood Recessive Diseases by Next-Generation Sequencing

Open access

Abstract

Of 7028 disorders with suspected Mendelian inheritance, 1139 are recessive and have an established molecular basis. Although individually uncommon, Mendelian diseases collectively account for ~20% of infant mortality and ~10% of pediatric hospitalizations. Preconception screening, together with genetic counseling of carriers, has resulted in remarkable declines in the incidence of several severe recessive diseases including Tay-Sachs disease and cystic fibrosis. However, extension of preconception screening to most severe disease genes has hitherto been impractical. Here, we report a preconception carrier screen for 448 severe recessive childhood diseases. Rather than costly, complete sequencing of the human genome, 7717 regions from 437 target genes were enriched by hybrid capture or microdroplet polymerase chain reaction, sequenced by next-generation sequencing (NGS) to a depth of up to 2.7 gigabases, and assessed with stringent bioinformatic filters. At a resultant 160x average target coverage, 93% of nucleotides had at least 20x coverage, and mutation detection/genotyping had ~95% sensitivity and ~100% specificity for substitution, insertion/deletion, splicing, and gross deletion mutations and single-nucleotide polymorphisms. In 104 unrelated DNA samples, the average genomic carrier burden for severe pediatric recessive mutations was 2.8 and ranged from 0 to 7. The distribution of mutations among sequenced samples appeared random. Twenty-seven percent of mutations cited in the literature were found to be common polymorphisms or misannotated, underscoring the need for better mutation databases as part of a comprehensive carrier testing strategy. Given the magnitude of carrier burden and the lower cost of testing compared to treating these conditions, carrier screening by NGS made available to the general population may be an economical way to reduce the incidence of and ameliorate suffering associated with severe recessive childhood disorders.

Overview

Integrity signals

Registry

Crossref · 10.1126/scitranslmed.3001756

Cites

64 of 64 references verified0 citation errors · 0 not checkable

Statistics

Nothing to recompute.

Verification

Claims the paper makes

50 Own result · 34 Own interpretation · 19 No basis given · 12 Partly supported · 9 Own method · 7 No full text of the sources · 5 Not checked yet · 4 Couldn’t confirm · 4 Sources not found · 2 Not supported · 1 Supported · accuracy not yet measured

Show all 71 claims

  • It may state two separate claims as one. 51
  • Probably not a substantial claim. 39
  • The quote only partly states this claim. 1
76 of 76
  • The authors state this as an assumption.

    Cost-effectiveness is critical for adoption of carrier testing.

    “First, cost-effectiveness was assumed to be critical for test adoption.”
  • The authors state this as an assumption.

    The incremental cost of increasing the degree of multiplexing decreases toward an asymptote.

    “The incremental cost associated with increasing the degree of multiplexing was assumed to decrease toward an asymptote.”
  • The authors state this as an assumption.

    Very broad coverage of diseases offers optimal cost-benefit.

    “Thus, very broad coverage of diseases was assumed to offer optimal cost-benefit.”
  • The authors state this as an assumption.

    Physician, patient, and societal adoption of screening is optimal for the most severe and highly penetrant childhood diseases, before conception, and where the anticipated clinical validity and utility of testing are clear.

    “Third, physician, patient, and societal adoption of screening was assumed to be optimal for the most severe and highly penetrant childhood diseases, before conception and where the anticipated clinical validity and clinical utility of testing was clear (26).”
  • The paper relies on this without support.

    The 104 unrelated DNA samples are representative of the general population for genomic carrier burden of severe pediatric recessive mutations.

    “In 104 unrelated DNA samples, the average genomic carrier burden for severe pediatric recessive mutations was 2.8 and ranged from 0 to 7.”
  • The paper relies on this without support.

    The cost of carrier screening by next-generation sequencing is lower than the cost of treating severe recessive childhood conditions.

    “Given the magnitude of carrier burden and the lower cost of testing compared to treating these conditions, carrier screening by NGS made available to the general population may be an economical way to reduce the incidence of and ameliorate suffering associated with severe recessive childhood disorders.”
  • The paper relies on this without support.

    Therapies are available for many severe recessive diseases of childhood.

    “Although TSD remains incurable, therapies are available for many severe recessive diseases of childhood.”
  • The paper relies on this without support.

    Specific national standards for quality assurance, quality control, test accessioning and reporting, and proficiency evaluation do not currently exist.

    “For example, specific national standards for quality assurance, quality control, test accessioning and reporting, and proficiency evaluation do not currently exist.”
  • The paper relies on this without support.

    A two-stage approach with confirmatory testing of all positive results is currently used for preconception carrier screening of couples.

    “Currently, a two-stage approach is used for preconception carrier screening of couples, with confirmatory testing of all positive results.”
  • The paper relies on this without support.

    Severe, orphan disease mutations have an incidence below 1% and are not found in the homozygous state in unaffected individuals.

    “Severe, orphan disease mutations should be uncommon (<1% incidence) and should not be found in the homozygous state in unaffected individuals.”
10 of 10

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

Each contradiction between two passages listed here was confirmed by an independent check.

4 of 4

Cited works that were retracted

Works this paper cites that were later retracted, from the retraction notices publishers deposit. Major when the notice came out before this paper was published.

1 of 1

Citations

Cites

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Metadata

Authors
Bell, C. J., Dinwiddie, D. L., Miller, N. A., Hateley, S. L., Ganusova, E. E., Mudge, J., Langley, R. J., Zhang, L., Lee, C. C., Schilkey, F. D., Sheth, V., Woodward, J. E., Peckham, H. E., Schroth, G. P., Kim, R. W., & Kingsmore, S. F.
Affiliations
  • National Center for Genome Resources (US)
  • Illumina (United States) (US)
  • Lifetech Scientific (China) (CN)
Corresponding author
  • Bell, Callum J.
  • Dinwiddie, Darrell L.
  • Kingsmore, Stephen F.
Venue
Science Translational Medicine
Published
2011-01-12
Volume
3
Issue
65
Pages
65ra4-65ra4
ISSN
  • 1946-6234 (ISSN-L)
  • 1946-6242
OpenAlex
W2064200890
Cited by
706
References
66
Citations by year
2026: 16 · 2025: 18 · 2024: 28 · 2023: 18 · 2022: 30 · 2021: 46 · 2020: 32 · 2019: 38 · 2018: 45 · 2017: 45 · 2016: 48 · 2015: 78 · 2014: 69 · 2013: 83 · 2012: 60
FWCI
51.97
Type
journal article
Language
en
Open access
Locations
  • Science Translational Medicine (publishedVersion)
  • PubMed (publishedVersion)
  • Europe PMC (PubMed Central) (acceptedVersion)
  • PubMed Central (acceptedVersion)
  • UNM’s Digital Repository (University of New Mexico) (submittedVersion)
Funders
  • National Institute of Allergy and Infectious Diseases (U01AI066569)
  • National Center for Research Resources (P20RR016480, RR 016480)
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (U19HD077693)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genomics and Rare Diseases
  • Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Prenatal Screening and Diagnostics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
Keywords
carrier screening, recessive genetic diseases, preconception screening, next-generation sequencing, targeted gene panel sequencing, hybridization capture, mutation detection, splicing mutation, large deletions, single nucleotide polymorphisms, mutation database, genetic counseling, Mendelian inheritance
MeSH terms
Base Sequence, Child, Female, Genes, Recessive, Genetic Testing, Heterozygote, Genetic Carrier Screening, Humans, Molecular Sequence Data, Mutation, Pregnancy, Prenatal Diagnosis, Genome, Human, Sequence Alignment, Sequence Analysis, DNA, Databases, Genetic

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