Evidence map›Paper›PMID 24299301›Full record

ArticleHuman gene therapy2014

OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy.

Mario Mietzsch, Sabrina Grasse, Catherine Zurawski, Stefan Weger, Antonette Bennett, Mavis Agbandje-McKenna, Nicholas Muzyczka, Sergei Zolotukhin, Regine Heilbronn

Abstract read
In one paragraph

Article in Human gene therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 87 papers.

0numbers the graph read from it
0cells of the map it votes in
87citing papers in PubMed
5.6field-weighted citation impact, top 4% of its field
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

87 citing papers in PubMed, 134 citations in OpenAlex.

  1. Article
  2. Article
  3. The amazing AAV capsids: Into the structure-verse.Molecular therapy. Methods & clinical development · 2025
    Review
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Review
  10. Article
  11. Article
  12. Article
  13. Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Review
  14. Article
  15. Article
  16. Clinical applications of gene therapy for rare diseases: A review.International journal of experimental pathology · 2023
    Review
  17. Article
  18. Article
  19. Viral Tools for Neural Circuit Tracing.Neuroscience bulletin · 2022
    Review
  20. Article

27 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

9 authors at 2 institutions in 3 countries.

Mario Mietzsch1 Institute of Virology , Campus Benjamin Franklin, Charité Universitätsmedizin Berlin, 12203 Berlin, Germany .
Sabrina Grasse
Catherine Zurawski
Stefan Weger
Antonette Bennett
Mavis Agbandje-McKenna
Nicholas Muzyczka
Sergei Zolotukhin
Regine Heilbronn
Institute of Virology of the Slovak Academy of Sciences · SKUniversity of Florida · US

Funding

Structural studies of AAV capsids and their glycan receptor interactionsR01GM082946 · NIGMS · UNIVERSITY OF FLORIDA · PI ROBERT MCKENNA · 2007 to 2026
$6.1M
NIAID NIH HHS NIH R01 AI1081961NIGMS NIH HHS R01 GM082946
6 · The paper itself

Abstract

Scalable and genetically stable recombinant adeno-associated virus (rAAV) production systems combined with facile adaptability for an extended repertoire of AAV serotypes are required to keep pace with the rapidly increasing clinical demand. For scalable high-titer production of the full range of rAAV serotypes 1-12, we developed OneBac, consisting of stable insect Sf9 cell lines harboring silent copies of AAV1-12 rep and cap genes induced upon infection with a single baculovirus that also carries the rAAV genome. rAAV burst sizes reach up to 5 × 10(5) benzonase-resistant, highly infectious genomic particles per cell, exceeding typical yields of current rAAV production systems. In contrast to recombinant rep/cap baculovirus strains currently employed for large-scale rAAV production, the Sf9rep/cap cell lines are genetically stable, leading to undiminished rAAV burst sizes over serial passages. Thus, OneBac combines full AAV serotype options with the capacity for stable scale-up production, the current bottleneck for the transition of AAV from gene therapy trials to routine clinical treatment.

Indexed as

AnimalsBaculoviridaeCell LineDependovirusGene Expression Regulation, ViralGenetic VectorsHEK293 CellsHeLa CellsHelper VirusesHumansTransduction, GeneticTransfectionViral ProteinsViral Proteins

Identifiers

PMID24299301
PMCPMC3955967
OpenAlexW1982076968

What OpenQuestion holds

Textmetadata
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.