Evidence map›Paper›PMID 31901636›Full record

ReviewiScience2020

Sharpening the Molecular Scissors: Advances in Gene-Editing Technology.

Mike Broeders, Pablo Herrero-Hernandez, Martijn P T Ernst, Ans T van der Ploeg, W W M Pim Pijnappel

Open access · goldAbstract readReview
In one paragraph

Review in iScience, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 61 papers.

0numbers the graph read from it
0cells of the map it votes in
61citing papers in PubMed
6.3field-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

61 citing papers in PubMed, 106 citations in OpenAlex.

  1. Review
  2. Frontiers in immunology · 2026
    Review
  3. Review
  4. Review
  5. Review
  6. Article
  7. Review
  8. Article
  9. Genomic Engineering of Induced Pluripotent Stem Cell-Derived Cardiomyocytes.Methods in molecular biology (Clifton, N.J.) · 2024
    Article
  10. Article
  11. Precision Genome Editing with CRISPR-Cas9.Methods in molecular biology (Clifton, N.J.) · 2024
    Article
  12. Review
  13. Review
  14. Review
  15. Artificial Olfactory Biohybrid System: An Evolving Sense of Smell.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2023
    Review
  16. CRISPR Gene Editing of Hematopoietic Stem and Progenitor Cells.Methods in molecular biology (Clifton, N.J.) · 2023
    Article
  17. Article
  18. Article
  19. Article
  20. Review

1 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

5 authors at 1 institution in 1 country.

Mike BroedersDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Department of Clinical Genetics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands.
Pablo Herrero-HernandezDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Department of Clinical Genetics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands.
Martijn P T ErnstDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Department of Clinical Genetics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands.
Ans T van der PloegDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands.
W W M Pim PijnappelDepartment of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Department of Clinical Genetics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands. Electronic address: w.pijnappel@erasmusmc.nl.
Erasmus MC · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The ability to precisely modify human genes has been made possible by the development of tools such as meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas. These now make it possible to generate targeted deletions, insertions, gene knock outs, and point variants; to modulate gene expression by targeting transcription factors or epigenetic machineries to DNA; or to target and modify RNA. Endogenous repair mechanisms are used to make the modifications required in DNA; they include non-homologous end joining, homology-directed repair, homology-independent targeted integration, microhomology-mediated end joining, base-excision repair, and mismatch repair. Off-target effects can be monitored using in silico prediction and sequencing and minimized using Cas proteins with higher accuracy, such as high-fidelity Cas9, enhanced-specificity Cas9, and hyperaccurate Cas9. Alternatives to Cas9 have been identified, including Cpf1, Cas12a, Cas12b, and smaller Cas9 orthologs such as CjCas9. Delivery of gene-editing components is performed ex vivo using standard techniques or in vivo using AAV, lipid nanoparticles, or cell-penetrating peptides. Clinical development of gene-editing technology is progressing in several fields, including immunotherapy in cancer treatment, antiviral therapy for HIV infection, and treatment of genetic disorders such as β-thalassemia, sickle cell disease, lysosomal storage disorders, and retinal dystrophy. Here we review these technological advances and the challenges to their clinical implementation.

Indexed as

GeneticsMolecular BiologyTechniques in Genetics

Identifiers

PMID31901636
PMCPMC6941877
OpenAlexW2995771391

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.