Evidence map›Paper›PMID 31945616›Full record

ReviewBiomaterials2020

Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications.

Yamin Li, Zachary Glass, Mingqian Huang, Zheng-Yi Chen, Qiaobing Xu

Abstract readReview
In one paragraph

Review in Biomaterials, 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
–field-weighted citation impact
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.

  1. Review
  2. The landscape and trajectory of global CRISPR therapeutics.Molecular therapy. Nucleic acids · 2026
    Review
  3. Review
  4. Review
  5. Article
  6. Review
  7. Review
  8. Review
  9. Review
  10. Landscape ofMolecular therapy. Methods & clinical development · 2025
    Article
  11. Review
  12. Article
  13. Article
  14. Review
  15. Journal of extracellular vesicles · 2025
    Article
  16. Article
  17. Review
  18. Review
  19. Review
  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.

Yamin LiDepartment of Biomedical Engineering, Tufts University, Medford, MA, 02155, USA.
Zachary GlassDepartment of Biomedical Engineering, Tufts University, Medford, MA, 02155, USA.
Mingqian HuangEaton-Peabody Laboratory, Massachusetts Eye and Ear, Department of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, 02114, USA.
Zheng-Yi ChenEaton-Peabody Laboratory, Massachusetts Eye and Ear, Department of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, 02114, USA. Electronic address: Zheng-Yi_Chen@meei.harvard.edu.
Qiaobing XuDepartment of Biomedical Engineering, Tufts University, Medford, MA, 02155, USA. Electronic address: Qiaobing.Xu@tufts.edu.

Funding

Development of Genome Editing as Treatment for Genetic Hearing LossR01DC016875 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI CHEN, ZHENG-YI · 2019 to 2023
$3.0M
Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid NanoparticlesUG3TR002636 · NCATS · MASSACHUSETTS EYE AND EAR INFIRMARY · PI CHEN, ZHENG-YI, LIU, DAVID R · 2018 to 2020
$2.4M
ROS-responsive Chemical Modification of Protein and Its DeliveryR01EB027170 · NIBIB · TUFTS UNIVERSITY MEDFORD · PI XU, QIAOBING · 2018 to 2022
$1.4M
NCATS NIH HHS UG3 TR002636NIBIB NIH HHS R01 EB027170NIDCD NIH HHS R01 DC016875
6 · The paper itself

Abstract

The recently developed CRISPR/Cas9 technology has revolutionized the genome engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics have entered clinical trials, most of which are focusing on the ex vivo genome editing. In this review, we highlight the ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. In these studies, CRISPR/Cas9 tools were used to edit cells in vitro and the successfully edited cells were considered as therapeutics, which can be introduced into patients to treat diseases. Considering a large number of previous reviews have been focused on the CRISPR/Cas9 delivery methods and materials, this review provides a different perspective, by mainly introducing the targeted conditions and design strategies for ex vivo CRISPR/Cas9 therapeutics. Brief descriptions of the history, functionality, and applications of CRISPR/Cas9 systems will be introduced first, followed by the design strategies and most significant results from previous research that used ex vivo CRISPR/Cas9 genome editing for the treatment of conditions or diseases. The last part of this review includes general information about the status of CRISPR/Cas9 therapeutics in clinical trials. We also discuss some of the challenges as well as the opportunities in this research area.

Indexed as

Clustered Regularly Interspaced Short Palindromic RepeatsGene EditingCRISPR-Cas SystemsGenetic TherapyGenomeHumansClinical trialsCRISPR/Cas9Ex vivoGenetic diseaseGenome editing

Identifiers

PMID31945616
PMCPMC7035593

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.