Evidence map›Paper›PMID 39139628›Full record

ArticleMolecular therapy. Methods & clinical development2024

A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations.

Noah K Taylor, Matthew J Guggenbiller, Pranali P Mistry, Oliver D King, Scott Q Harper

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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

12 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Review
  5. Article
  6. Review
  7. Residual DNA impurities in AAV vectors-nature and transcription.Molecular therapy. Methods & clinical development · 2025
    Article
  8. Article
  9. AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies.International journal of molecular sciences · 2025
    Review
  10. Review
  11. Review
  12. 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

5 authors.

Noah K TaylorCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43215, USA.
Matthew J GuggenbillerCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43215, USA.
Pranali P MistryCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43215, USA.
Oliver D KingDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Scott Q HarperCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43215, USA.

Funding

CRISPR-Cas13 gene therapy and RNA editing for Facioscapulohumeral muscular dystrophy (FSHD)R01AR078395 · NIAMS · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI HARPER, SCOTT Q · 2021 to 2025
$2.9M
NIAMS NIH HHS R01 AR078395
6 · The paper itself

Abstract

Adeno-associated viral vectors (AAVs) are a leading delivery system for gene therapy in animal models and humans. With several Food and Drug Administration-approved AAV gene therapies on the market, issues related to vector manufacturing have become increasingly important. In this study, we focused on potentially toxic DNA contaminants that can arise from AAV proviral plasmids, the raw materials required for manufacturing recombinant AAV in eukaryotic cells. Typical AAV proviral plasmids are circular DNAs containing a therapeutic gene cassette flanked by natural AAV inverted terminal repeat (ITR) sequences, and a plasmid backbone carrying prokaryotic sequences required for plasmid replication and selection in bacteria. While the majority of AAV particles package the intended therapeutic payload, some capsids instead package the bacterial sequences located on the proviral plasmid backbone. Since ITR sequences also have promoter activity, potentially toxic bacterial open reading frames can be produced

Indexed as

AAVAAV biologyAAV cross-packagingAAV transfer plasmidadeno-associated virusCTCFinsulatorsinverted terminal repeatvector engineering

Identifiers

PMID39139628
PMCPMC11320455

What OpenQuestion holds

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Registered trials

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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.