Evidence map›Paper›PMID 39690326›Full record

ReviewNature reviews. Drug discovery2025

Chemical engineering of CRISPR-Cas systems for therapeutic application.

Halle M Barber, Adrian A Pater, Keith T Gagnon, Masad J Damha, Daniel O'Reilly

Abstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing 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

25 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Article
  5. Review
  6. Review
  7. Review
  8. SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  9. Article
  10. Review
  11. Article
  12. Article
  13. Review
  14. Review
  15. Synergistic Interactions of Anthraquinones with Conventional Cancer Therapies.Combinatorial chemistry & high throughput screening · 2026
    Review
  16. A guide RNA repeat checkpoint steers CRISPR-Cas9 catalysis.bioRxiv : the preprint server for biology · 2025
    Article
  17. Article
  18. RNA chemistry and therapeutics.Nature reviews. Drug discovery · 2025
    Review
  19. Article
  20. Chemically modified non-coding RNAs in cancer.Expert reviews in molecular medicine · 2025
    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.

Halle M Barber *Department of Chemistry, McGill University, Montreal, Quebec, Canada.ORCID 0000-0003-3514-7240
Adrian A Pater *Department of Biochemistry, Wake Forest University School of Medicine, Winston-Salem, NC, USA.
Keith T GagnonDepartment of Biochemistry, Wake Forest University School of Medicine, Winston-Salem, NC, USA. ktgagnon@wakehealth.edu.ORCID 0000-0002-5868-675X
Masad J DamhaDepartment of Chemistry, McGill University, Montreal, Quebec, Canada. masad.damha@mcgill.ca.ORCID 0000-0002-4458-1623
Daniel O'ReillyDepartment of Pharmacology and Toxicology, University of Texas Medical Branch, Galveston, TX, USA. dtoreill@utmb.edu.ORCID 0000-0003-0012-5623

Funding

Nucleic Acid-Based Anti-CRISPR Inhibitors of Cas9R01GM135646 · NIGMS · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI GAGNON, KEITH THOMAS · 2020 to 2023
$1.1M
NIGMS NIH HHS R01 GM135646
6 · The paper itself

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) technology has transformed molecular biology and the future of gene-targeted therapeutics. CRISPR systems comprise a CRISPR-associated (Cas) endonuclease and a guide RNA (gRNA) that can be programmed to guide sequence-specific binding, cleavage, or modification of complementary DNA or RNA. However, the application of CRISPR-based therapeutics is challenged by factors such as molecular size, prokaryotic or phage origins, and an essential gRNA cofactor requirement, which impact efficacy, delivery and safety. This Review focuses on chemical modification and engineering approaches for gRNAs to enhance or enable CRISPR-based therapeutics, emphasizing Cas9 and Cas12a as therapeutic paradigms. Issues that chemically modified gRNAs seek to address, including drug delivery, physiological stability, editing efficiency and off-target effects, as well as challenges that remain, are discussed.

Indexed as

Chemical EngineeringClustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsGene EditingGenetic TherapyRNA, Guide, CRISPR-Cas SystemsAnimalsDrug Delivery SystemsHumansRNA, Guide, CRISPR-Cas Systems

Identifiers

PMID39690326
PMCPMC13347398

What OpenQuestion holds

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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.