Evidence map›Paper›PMID 24685129›Full record

ArticleCell reports2014

Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing.

Ayal Hendel, Eric J Kildebeck, Eli J Fine, Joseph Clark, Niraj Punjya, Vittorio Sebastiano, Gang Bao, Matthew H Porteus

Open access · goldAbstract read
In one paragraph

Article in Cell reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 79 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
79citing papers in PubMed, 1 pooled it
7.7field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

79 citing papers in PubMed, 1 synthesis or guideline pooled it, 133 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. Review
  6. Genome engineering with Cas9 and AAV repair templates, successes and pitfalls.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review
  7. Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. The p53 challenge of hematopoietic stem cell gene editing.Molecular therapy. Methods & clinical development · 2023
    Review
  13. Article
  14. Review
  15. Article
  16. Whole Genome Sequencing Analysis of Effects of CRISPR/Cas9 inJournal of fungi (Basel, Switzerland) · 2022
    Article
  17. Review
  18. Article
  19. Article
  20. Article

19 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 2 institutions in 1 country.

Ayal Hendel *Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Eric J Kildebeck *Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Eli J Fine *Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA 30332, USA.
Joseph ClarkDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Niraj PunjyaDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Vittorio SebastianoDepartment of Obstetrics and Gynecology, Stanford University, Stanford, CA 94305, USA.
Gang BaoDepartment of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA 30332, USA.
Matthew H PorteusDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Stanford University · USEmory University · US

Funding

Nanomedicine Center for Nucleoprotein MachinesPN2EY018244 · NEI · RICE UNIVERSITY · PI BAO, GANG · 2006 to 2015
$24.4M
Pre-Clinical Development of Nuclease Mediated Gene Therapy for SCIDR01AI097320 · NIAID · STANFORD UNIVERSITY · PI PORTEUS, MATTHEW H · 2013 to 2017
$1.9M
NEI NIH HHS PN2 EY018244NEI NIH HHS PN2EY018244NIAID NIH HHS R01 AI097320
6 · The paper itself

Abstract

Targeted genome editing with engineered nucleases has transformed the ability to introduce precise sequence modifications at almost any site within the genome. A major obstacle to probing the efficiency and consequences of genome editing is that no existing method enables the frequency of different editing events to be simultaneously measured across a cell population at any endogenous genomic locus. We have developed a method for quantifying individual genome-editing outcomes at any site of interest with single-molecule real-time (SMRT) DNA sequencing. We show that this approach can be applied at various loci using multiple engineered nuclease platforms, including transcription-activator-like effector nucleases (TALENs), RNA-guided endonucleases (CRISPR/Cas9), and zinc finger nucleases (ZFNs), and in different cell lines to identify conditions and strategies in which the desired engineering outcome has occurred. This approach offers a technique for studying double-strand break repair, facilitates the evaluation of gene-editing technologies, and permits sensitive quantification of editing outcomes in almost every experimental system used.

Indexed as

Genome, HumanRepetitive Sequences, Nucleic AcidDNA Breaks, Double-StrandedDNA RepairEndonucleasesGenetic EngineeringHumansK562 CellsSequence Analysis, DNATransfectionEndonucleases

Identifiers

PMID24685129
PMCPMC4015468
OpenAlexW2018456465

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.