Evidence map›Paper›PMID 30169977›Full record

ReviewExpert opinion on drug delivery2018

Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges.

D C Luther, Y W Lee, H Nagaraj, F Scaletti, V M Rotello

Abstract readReview
In one paragraph

Review in Expert opinion on drug delivery, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 81 papers.

0numbers the graph read from it
0cells of the map it votes in
81citing 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

81 citing papers in PubMed.

  1. Review
  2. CRISPR/Cas9-based nanodelivery systems for precision gene editing in neuroinflammation.Inflammation research : official journal of the European Histamine Research Society ... [et al.] · 2026
    Review
  3. Article
  4. Review
  5. Article
  6. Review
  7. Article
  8. Review
  9. The emerging metabolic role and treatment target of CPT1A in CRC.Frontiers in cell and developmental biology · 2026
    Review
  10. Article
  11. Detection of CRISPR-Cas-induced mutations inbioRxiv : the preprint server for biology · 2025
    Article
  12. Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. Review

21 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.

D C Luthera Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.
Y W Leea Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.
H Nagaraja Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.
F Scalettia Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.
V M Rotelloa Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.

Funding

CHEMISTRY-BIOLOGY INTERFACE PREDOCTORAL TRAINING GRANTST32GM008515 · NIGMS · UNIVERSITY OF MASSACHUSETTS AMHERST · PI THOMPSON, LYNMARIE K. · 1995 to 2020
$4.3M
Recognition and Presentation of Alpha Helices using Nanoparticle ReceptorsR01GM077173 · NIGMS · UNIVERSITY OF MASSACHUSETTS AMHERST · PI ROTELLO, VINCENT M. · 2007 to 2018
$3.7M
Targeting of Bio-orthogonal Chemotherapeutic Nanozymes to Tumor-Associated MacrophagesR01EB022641 · NIBIB · UNIVERSITY OF MASSACHUSETTS AMHERST · PI ROTELLO, VINCENT M. · 2017 to 2024
$3.0M
NIBIB NIH HHS R01 EB022641NIGMS NIH HHS R01 GM077173NIGMS NIH HHS T32 GM008515
6 · The paper itself

Abstract

introductionTherapeutic gene editing is becoming a viable biomedical tool with the emergence of the CRISPR/Cas9 system. CRISPR-based technologies have promise as a therapeutic platform for many human genetic diseases previously considered untreatable, providing a flexible approach to high-fidelity gene editing. For many diseases, such as sickle-cell disease and beta thalassemia, curative therapy may already be on the horizon, with CRISPR-based clinical trials slated for the next few years. Translation of CRISPR-based therapy to in vivo application however, is no small feat, and major hurdles remain for efficacious use of the CRISPR/Cas9 system in clinical contexts. AREAS COVERED: In this topical review, we highlight recent advances to in vivo delivery of the CRISPR/Cas9 system using various packaging formats, including viral, mRNA, plasmid, and protein-based approaches. We also discuss some of the barriers which have yet to be overcome for successful translation of this technology. EXPERT OPINION: This review focuses on the challenges to efficacy for various delivery formats, with specific emphasis on overcoming these challenges through the development of carrier vehicles for transient approaches to CRISPR/Cas9 delivery in vivo.

Indexed as

AnimalsClustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsGene EditingGenetic TherapyGene Transfer TechniquesHumansRNA, MessengerRNA, MessengerCRISPR/Cas9gene therapyintracellular deliveryin vivo genome editingnonviral deliverytherapeutic strategiesviral delivery

Identifiers

PMID30169977
PMCPMC6295289

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.