Cited by 427Genomics and Chromatin DynamicsChromatin Remodeling and CancerGenetic Associations and Epidemiology

The DOI matches its Crossref record; 255 of the 255 references it cites were found in a registry or an index; 133 of the 157 sources its claims cite were checked for support.

Towards a comprehensive catalogue of validated and target-linked human enhancers

Open access
Gasperini, Molly, Tome, Jacob M., Shendure, Jay·2020·Nature Reviews Genetics

Abstract

The human gene catalogue is essentially complete, but we lack an equivalently vetted inventory of bona fide human enhancers. Hundreds of thousands of candidate enhancers have been nominated via biochemical annotations; however, only a handful of these have been validated and confidently linked to their target genes. Here we review emerging technologies for discovering, characterizing and validating human enhancers at scale. We furthermore propose a new framework for operationally defining enhancers that accommodates the heterogeneous and complementary results that are emerging from reporter assays, biochemical measurements and CRISPR screens.

Overview

AI snapshot

Written from the paper’s full text.

TL;DR

This review argues that the human enhancer catalogue remains far from complete: over one million candidate cis-regulatory elements have been annotated biochemically, but only a handful are validated and linked to their target genes. It surveys scalable biochemical, reporter, CRISPR, and in vivo methods and proposes a tiered framework for calling enhancers validated and target-linked, strongly, moderately, or weakly supported.

Key findings

  • Biochemical annotations have catalogued over one million candidate cis-regulatory elements with enhancer-like signatures, collectively spanning ~16% of the human genome, but these are not equivalent to validated enhancers and often lack target-gene links.
  • In a whole-transcriptome CRISPRi screen (REF72), ~90% of tested biochemically or MPRA-supported candidate enhancers did not detectably influence expression of a cis-encoded gene; possible explanations include technical false negatives, shadow enhancers, stable cell lines masking regulatory effects, or fewer bona fide enhancers than annotations suggest.
  • Emerging methods include MPRAs, CRISPR/CRISPRi/CRISPRa screens in native context, single-cell profiling, 3D conformation mapping, eQTLs, and mouse validation; one multiplexed scRNA-seq CRISPRi experiment evaluated 5,779 candidate enhancers with ~28 gRNAs per cell, while the first such screen targeted 71 candidate enhancers across 7 genomic loci.
  • The proposed framework defines “validated and target-linked” enhancers as requiring targeted deletion that alters a distal target gene, evidence for a cis-acting mechanism, and at least one orthogonal enhancer line of evidence (episomal reporter activity or enhancer-associated biochemical marks); lower tiers are strongly supported (3/3 evidence classes), moderately supported (2/3), and weakly supported (1/3).

Methods

  • Design: Review/survey, not a primary experimental study; it synthesizes the history of enhancer concepts, current and emerging technologies, and proposes an evidentiary framework.
  • Data: Published literature and existing datasets, including ENCODE biochemical annotations, MPRAs, CRISPR/CRISPRi/CRISPRa screens, eQTL studies, 3C/Hi-C and single-cell methods, and mouse transgenic reporter or CRISPR deletion assays.
  • Analysis: Narrative synthesis and conceptual framework development; no new data collection or statistical analysis is reported.

Limitations

  • The paper states that operational definitions of enhancers—reporter-based, biochemical, and biological—are not equivalent; biochemical annotations can come from derived or mixed cell lines, may not demonstrate cis regulation, fail to specify target genes, and may not reflect mechanism; MPRAs are out-of-context, often use a single promoter and episomes; CRISPR screens are limited by effect size, efficiency, NHEJ variability, heterogeneous allele editing, and unknown false-negative rates; and it is impractical to prove a sequence is not an enhancer.
  • The paper states that different assay types frequently disagree and that the sensitivity and specificity of most assays remain largely unknown; the proposed framework is also described as light in detail and may not adapt easily to enhancers overlapping promoters or protein-coding regions.
  • Evident from the text: as a review, it reports no new experimental validation of the proposed tiers; the framework is a proposal, not an empirically tested classification scheme.

Model-written summary. Check it against the paper before relying on it.

Integrity signals

Registry

Crossref · 10.1038/s41576-019-0209-0

Cites

255 of 255 references verified0 citation errors · 0 not checkable

Statistics

Nothing to recompute.

Verification

Claims the paper makes

61 Own interpretation · 38 Partly supported · 20 Supported · 9 No basis given · 8 No full text of the sources · 4 Supported so far · 2 Couldn’t confirm · 1 Mixed support · 1 Not supported · 1 Sources not found · accuracy not yet measured

Show all 62 claims

  • Probably not a substantial claim. 52
  • It may state two separate claims as one. 47
83 of 83
  • The paper relies on this without support.

    The human gene catalogue is essentially complete.

    “The human gene catalogue is essentially complete, but we lack an equivalently vetted inventory of bona fide human enhancers.”
  • The authors state this as an assumption.

    Enhancers are the most numerically prevalent class of non-coding DNA regulatory element.

    “Enhancers are only one class of non-coding DNA regulatory element, although they are widely presumed to be the most numerically prevalent (Box 2).”
  • The authors state this as an assumption.

    The greater complexity of mammalian development is encoded by enhancers.

    “If the greater complexity of mammalian development was instead encoded by enhancers, a belief that took hold at the time and persists today, they too required cataloguing and characterization.”
  • The paper relies on this without support.

    Most enhancers do not harbour common variants that substantially perturb their activity.

    “On the other hand, the framework has clear limitations, including its reliance on naturally occurring human genetic variation (most enhancers do not harbour common variants that substantially perturb their activity), linkage disequilibrium [G] (multiple variants in a haplotype block may equivalently explain an association), and restriction to cell types and tissues that can be practically obtained”
  • The authors state this as an assumption.

    The human genome harbours hundreds-of-thousands to millions of enhancers.

    “The human genome is currently believed to harbour hundreds-of-thousands to millions of enhancers — stretches of DNA that bind transcription factors [G] (TFs) and enhance the expression of genes encoded in cis.”
  • The authors state this as an assumption.

    Enhancers play a principal role in orchestrating the gene expression program underlying human development and homeostasis.

    “Collectively, enhancers are thought to play a principal role in orchestrating the fantastically complex program of gene expression that underlies human development and homeostasis.”
  • The paper relies on this without support.

    Individual enhancers most likely only regulate one or a few genes in cis.

    “As scRNA-seq is costly and individual enhancers most likely only regulate one or a few genes in cis, we developed a related approach wherein ~28 gRNAs were introduced per cell, enabling 5,779 candidate enhancers to be evaluated in a single experiment72.”
  • The paper relies on this without support.

    Perturbations of non-coding elements can have secondary effects.

    “Perturbations of non-coding elements can have secondary effects, so there should be at least some rationale for concluding that an observed effect is mediated primarily by a cis regulatory mechanism.”
  • The paper relies on this without support.

    It is very difficult to prove a sequence is not an enhancer.

    “As discussed above, it is very difficult to prove a sequence is not an enhancer.”
  • The paper relies on this without support.

    These assays (biochemical marks, episomal reporter activity, and CRISPR perturbation) are correlated.

    “Of course, these assays are correlated, so in many cases there will be agreement across the board.”
10 of 10

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

1 of 1

Citations

Cites

Research works printed but not linked: 1

  1. Benabdallah, N. S. et al. Decreased enhancer–promoter proximity accompanying enhancer activation. Mol. Cell 76, 473–484.e7 (2019). · DOI · Paper page

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Metadata

Authors
Gasperini, M., Tome, J. M., & Shendure, J.
Affiliations
  • University of Washington (US)
  • Howard Hughes Medical Institute (US)
Corresponding author
Shendure, Jay
Venue
Nature Reviews Genetics
Published
2020-01-27
Volume
21
Issue
5
Pages
292-310
ISSN
  • 1471-0056 (ISSN-L)
  • 1471-0064
OpenAlex
W3000799818
Cited by
396
References
248
Citations by year
2026: 36 · 2025: 55 · 2024: 84 · 2023: 65 · 2022: 51 · 2021: 75 · 2020: 29
FWCI
5.35
Type
journal article
Language
en
Open access
Locations
  • Nature Reviews Genetics (publishedVersion)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
Funders
National Human Genome Research Institute (UM1 HG009408, T32HG000035)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Genomics and Chromatin Dynamics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Chromatin Remodeling and Cancer
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic Associations and Epidemiology
Keywords
reporter assay, CRISPR screen, transcriptional regulation
MeSH terms
Enhancer Elements, Genetic, Humans, Genome, Human, Clustered Regularly Interspaced Short Palindromic Repeats

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