Evidence map›Paper›PMID 36334354›Full record

ReviewBiomaterials2022

In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications.

Kun Huang, Daniel Zapata, Yan Tang, Yong Teng, Yamin Li

Open access · greenAbstract readReview
In one paragraph

Review in Biomaterials, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
3.8field-weighted citation impact, top 5% 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

26 citing papers in PubMed, 47 citations in OpenAlex.

  1. Overview of Delivery Methods for Gene Editing.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  2. Review
  3. In Vivo T-Cell Engineering: Revolution in Delivery Strategies and Clinical Translation.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  4. Article
  5. [Emphasizing evolution of key technological elements in reconstructive surgery].Zhongguo xiu fu chong jian wai ke za zhi = Zhongguo xiufu chongjian waike zazhi = Chinese journal of reparative and reconstructive surgery · 2026
    Review
  6. International journal of biological sciences · 2026
    Review
  7. Review
  8. Review
  9. Article
  10. Review
  11. Review
  12. Review
  13. Review
  14. Targeted Human Editing Using CRISPR Technology in Neural Stem Cells.Methods in molecular biology (Clifton, N.J.) · 2025
    Article
  15. Article
  16. In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  17. CRISPR-Cas System: A New Dawn to Combat Antibiotic Resistance.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024
    Review
  18. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.International journal of molecular sciences · 2024
    Review
  19. Heliyon · 2024
    Review
  20. Review
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 3 institutions in 1 country.

Kun HuangDepartment of Pharmacology, State University of New York, Upstate Medical University, Syracuse, NY, 13210, USA.
Daniel ZapataDepartment of Pharmacology, State University of New York, Upstate Medical University, Syracuse, NY, 13210, USA.
Yan TangDivision of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, 02115, USA.
Yong TengDepartment of Hematology and Medical Oncology, Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA, 30322, USA. Electronic address: yong.teng@emory.edu.
Yamin LiDepartment of Pharmacology, State University of New York, Upstate Medical University, Syracuse, NY, 13210, USA. Electronic address: liyam@upstate.edu.
SUNY Upstate Medical University · USBrigham and Women's Hospital · USEmory University · US

Funding

ATAD3A as a Novel Target in Head and Neck CancerR01DE028351 · NIDCR · EMORY UNIVERSITY · PI TENG, YONG · 2019 to 2023
$1.9M
Role of FAT1 somatic mutations in aggressiveness of head and neck cancerR03DE032084 · NIDCR · EMORY UNIVERSITY · PI CHEN, ZHENGJIA, TENG, YONG · 2022 to 2023
$328k
The Impact of Arf1 on Hypoxia Signaling and Oral CancerR03DE028387 · NIDCR · EMORY UNIVERSITY · PI TENG, YONG · 2019 to 2020
$306k
NIDCR NIH HHS R01 DE028351NIDCR NIH HHS R03 DE028387NIDCR NIH HHS R03 DE032084
6 · The paper itself

Abstract

Since its mechanism discovery in 2012 and the first application for mammalian genome editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created countless opportunities in both basic science and translational medicine. The first clinical trial of CRISPR therapeutics was initiated in 2016, which employed ex vivo CRISPR-Cas9 edited PD-1 knockout T cells for the treatment of non-small cell lung cancer. So far there have been dozens of clinical trials registered on ClinicalTrials.gov in regard to using the CRISPR-Cas9 genome editing as the main intervention for therapeutic applications; however, most of these studies use ex vivo genome editing approach, and only a few apply the in vivo editing strategy. Compared to ex vivo editing, in vivo genome editing bypasses tedious procedures related to cell isolation, maintenance, selection, and transplantation. It is also applicable to a wide range of diseases and disorders. The main obstacles to the successful translation of in vivo therapeutic genome editing include the lack of safe and efficient delivery system and safety concerns resulting from the off-target effects. In this review, we highlight the therapeutic applications of in vivo genome editing mediated by the CRISPR-Cas9 system. Following a brief introduction of the history, biology, and functionality of CRISPR-Cas9, we showcase a series of exemplary studies in regard to the design and implementation of in vivo genome editing systems that target the brain, inner ear, eye, heart, liver, lung, muscle, skin, immune system, and tumor. Current challenges and opportunities in the field of CRISPR-enabled therapeutic in vivo genome editing are also discussed.

Indexed as

Carcinoma, Non-Small-Cell LungLung NeoplasmsAnimalsCRISPR-Cas SystemsGene EditingGene Transfer TechniquesHumansMammalsCRISPR-Cas9Genome editingNanomaterialOrgan targetingViral vector

Identifiers

PMID36334354
PMCPMC10018374
OpenAlexW4307437981

What OpenQuestion holds

Textmetadata
LicenceTDM
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.