Evidence map›Paper›PMID 38437567›Full record

ReviewProceedings of the National Academy of Sciences of the United States of America2024

Targeted nonviral delivery of genome editors in vivo.

Connor A Tsuchida, Kevin M Wasko, Jennifer R Hamilton, Jennifer A Doudna

Abstract readReview
In one paragraph

Review in Proceedings of the National Academy of Sciences of the United States of America, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 57 papers.

0numbers the graph read from it
0cells of the map it votes in
57citing 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

57 citing papers in PubMed.

  1. Overview of Delivery Methods for Gene Editing.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  2. Review
  3. Review
  4. Towards mRNA therapeutics 2.0.Nature reviews. Drug discovery · 2026
    Review
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  11. Article
  12. Review
  13. 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
  14. Review
  15. Article
  16. Article
  17. Article
  18. Article
  19. Review
  20. Article
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

4 authors.

Connor A Tsuchida *University of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, CA 94720.ORCID 0000-0002-9563-8052
Kevin M Wasko *Innovative Genomics Institute, University of California, Berkeley, CA 94720.
Jennifer R Hamilton *Innovative Genomics Institute, University of California, Berkeley, CA 94720.ORCID 0000-0002-7136-1757
Jennifer A DoudnaUniversity of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, CA 94720.ORCID 0000-0001-9161-999X

Funding

Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
Expanding CRISPR-Cas editing technology through exploration of novel Cas proteins and DNA repair systemsU01AI142817 · NIAID · UNIVERSITY OF CALIFORNIA BERKELEY · PI BANFIELD, JILLIAN, DOUDNA, JENNIFER A · 2018 to 2022
$2.0M
Uncovering cell-intrinsic restrictions to CRISPR-Cas9 gene editingK99GM143461 · NIGMS · UNIVERSITY OF CALIFORNIA BERKELEY · PI HAMILTON, JENNIFER R · 2022 to 2023
$214k
Characterization and engineering of a compact genome editor for adeno-associated virus delivery to correct mutations causing cystic fibrosisF31HL156468 · NHLBI · UNIVERSITY OF CALIFORNIA BERKELEY · PI TSUCHIDA, CONNOR ANDREW · 2021 to 2022
$77k
NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS F31 HL156468NIAID NIH HHS U01 AI142817NIGMS NIH HHS K99 GM143461
6 · The paper itself

Abstract

Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that will drive biological discovery and transform the field of cell and gene therapy. Here, we discuss recent advances in the delivery of CRISPR-Cas genome editors either as preassembled ribonucleoproteins or encoded in mRNA. Both strategies avoid pitfalls of viral vector-mediated delivery and offer advantages including transient editor lifetime and potentially streamlined manufacturing capability that are already proving valuable for clinical use. We review current applications and future opportunities of these emerging delivery approaches that could make genome editing more efficacious and accessible in the future.

Indexed as

CommerceGene EditingGenetic TherapyRibonucleoproteinsRNA, MessengerRibonucleoproteinsRNA, MessengerCRISPR-Casgenome editingin vivo deliverynonviral deliverytargeted delivery

Identifiers

PMID38437567
PMCPMC10945750

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.