Evidence map›Paper›PMID 30984891›Full record

ArticleCurrent opinion in biomedical engineering2018

Debugging the genetic code: non-viral

Alexandra S Piotrowski-Daspit, Peter M Glaze, W Mark Saltzman

Abstract read
In one paragraph

Article in Current opinion in biomedical engineering, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing 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

11 citing papers in PubMed.

  1. Review
  2. Engineered cargo-free nanoparticle decoys for phagocytic modulation of macrophages.Journal of controlled release : official journal of the Controlled Release Society · 2026
    Article
  3. Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Review
  9. In vivo somatic cell base editing and prime editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  10. Article
  11. Polymeric vehicles for nucleic acid delivery.Advanced drug delivery reviews · 2020
    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

3 authors.

Alexandra S Piotrowski-DaspitDepartment of Biomedical Engineering, Yale University, New Haven, CT 06511.
Peter M GlazeDepartment of Therapeutic Radiology, Yale School of Medicine, New Haven, CT 06510.
W Mark SaltzmanDepartment of Biomedical Engineering, Yale University, New Haven, CT 06511.

Funding

Research Training in AnesthesiaT32GM086287 · NIGMS · YALE UNIVERSITY · PI ROBERT SCHONBERGER · 2009 to 2026
$3.7M
Targeted correction of the human CFTR geneR01HL125892 · NHLBI · YALE UNIVERSITY · PI EGAN, MARIE E, GLAZER, PETER M · 2015 to 2018
$2.2M
In vivo genomic editing of hematopoietic cells for HIV resistanceR01AI112443 · NIAID · YALE UNIVERSITY · PI GLAZER, PETER M, KUMAR, PRITI · 2014 to 2017
$2.1M
Developing Poly(amine-co-ester) Polymeric Delivery Vehicles and 3D Culture Screening Platforms for the Treatment of Hereditary Beta-thalassemia with Peptide Nucleic Acid Genome Editing TechnologyF32HL142144 · NHLBI · YALE UNIVERSITY · PI PIOTROWSKI-DASPIT, ALEXANDRA SARAH ANNUKKA · 2018 to 2019
$123k
NHLBI NIH HHS F32 HL142144NHLBI NIH HHS R01 HL125892NIAID NIH HHS R01 AI112443NIGMS NIH HHS T32 GM086287
6 · The paper itself

Abstract

Efforts to precisely correct genomic mutations that underlie hereditary diseases for therapeutic benefit have advanced alongside the emergence and improvement of genome engineering technologies. These methods can be divided into two main classes: active nucleasebased platforms including the popular CRISPR/Cas9 system and oligo/polynucleotide strategies including triplex-forming oligonucleotides (TFOs), such as peptide nucleic acids (PNAs). These technologies have been successful in cell culture and in animals, but important challenges remain before these tools can be translated into the clinic; they must be effectively delivered to and taken up by specific cell types of interest, achieve correction levels in target cells that significantly ameliorate the disease phenotype, and demonstrate minimal off-target and toxicity effects. Here we review and compare the current strategies and non-viral delivery methods, mainly lipid and polymeric vehicles, proposed for genome editing of inherited disorders with a focus on

Indexed as

CRISPR/Cas9non-viral deliverynuclease-mediated genome editingoligonucleotide-mediated genome editingpeptide nucleic acidtherapeutic genome editing

Identifiers

PMID30984891
PMCPMC6456264

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.