Evidence map›Paper›PMID 31530236›Full record

ArticleHuman gene therapy2019

Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy.

Suriyasri Subramanian, Anna C Maurer, Carol M Bator, Alexander M Makhov, James F Conway, Kevin B Turner, James H Marden, Luk H Vandenberghe, Susan L Hafenstein

Open access · greenAbstract read
In one paragraph

Article in Human gene therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
2.7field-weighted citation impact, top 9% 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

23 citing papers in PubMed, 40 citations in OpenAlex.

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  7. Review
  8. The myth of high-resolution liquid phase biological electron microscopy.Protein science : a publication of the Protein Society · 2024
    Article
  9. Article
  10. Article
  11. Integrative Approaches to Study Virus Structures.Sub-cellular biochemistry · 2024
    Review
  12. Article
  13. Article
  14. Article
  15. Article
  16. Review
  17. Article
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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 4 institutions in 1 country.

Suriyasri SubramanianDepartment of Medicine, The Pennsylvania State University College of Medicine, Hershey, Pennsylvania.
Anna C MaurerGrousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear, Boston, Massachusetts.
Carol M BatorHuck Institutes of the Life Sciences, Pennsylvania State University, University Park, Pennsylvania.
Alexander M MakhovDepartment of Structural Biology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
James F ConwayDepartment of Structural Biology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
Kevin B TurnerGene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
James H MardenHuck Institutes of the Life Sciences, Pennsylvania State University, University Park, Pennsylvania.
Luk H VandenbergheGrousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear, Boston, Massachusetts.
Susan L HafensteinDepartment of Medicine, The Pennsylvania State University College of Medicine, Hershey, Pennsylvania.
Pennsylvania State University · USBroad Institute · USUniversity of Pittsburgh · USUniversity of Pennsylvania · US

Funding

Direct electron detecting (DED) cameraS10OD019995 · OD · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI CONWAY, JAMES F. · 2015 to 2015
$548k
NIH HHS S10 OD019995
6 · The paper itself

Abstract

Adeno-associated viruses (AAVs) have been employed successfully as gene therapy vectors in treating various genetic diseases for almost two decades. However, transgene packaging is usually imperfect, and developing a rapid and accurate method for measuring the proportion of DNA encapsidation is an important step for improving the downstream process of large scale vector production. In this study, we used two-dimensional class averages and three-dimensional classes, intermediate outputs in the single particle cryo-electron microscopy (cryo-EM) image reconstruction pipeline, to determine the proportion of DNA-packaged and empty capsid populations. Two different preparations of AAV3 were analyzed to estimate the minimum number of particles required to be sampled by cryo-EM in order for robust calculation of the proportion of the full versus empty capsids in any given sample. Cost analysis applied to the minimum amount of data required for a valid ratio suggests that cryo-EM is an effective approach to analyze vector preparations.

Indexed as

Cryoelectron MicroscopyCapsidCapsid ProteinsDependovirusGenetic VectorsHumansVirionCapsid Proteins2D classesAAVcryo-EMDNA filledpercentage filled

Identifiers

PMID31530236
PMCPMC6921155
OpenAlexW2987565474

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.