Evidence map›Paper›PMID 28427840›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017

AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.

Simon Pacouret, Mohammed Bouzelha, Rajani Shelke, Eva Andres-Mateos, Ru Xiao, Anna Maurer, Mathieu Mevel, Heikki Turunen, Trisha Barungi, Magalie Penaud-Budloo and 5 more

Open access · bronzeAbstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed
2.5field-weighted citation impact, top 10% 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

41 citing papers in PubMed, 66 citations in OpenAlex.

  1. Review
  2. Article
  3. Review
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
  13. Review
  14. Article
  15. Cryo-EM structure of adeno-associated virus 4 at 2.2 Å resolution.Acta crystallographica. Section D, Structural biology · 2023
    Article
  16. Article
  17. Article
  18. Article
  19. Review
  20. 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

15 authors at 4 institutions in 2 countries.

Simon PacouretGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA; Atlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Mohammed BouzelhaAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Rajani ShelkeGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA.
Eva Andres-MateosGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA.
Ru XiaoGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA.
Anna MaurerGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA; Biological and Biomedical Sciences Program, Division of Medical Sciences, Harvard Medical School, Boston, MA 02115, USA.
Mathieu MevelAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Heikki TurunenGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA.
Trisha BarungiGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA.
Magalie Penaud-BudlooAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Frédéric BroucqueAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Véronique BlouinAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Philippe MoullierAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Eduard AyusoAtlantic Gene Therapies, INSERM UMR 1089, University of Nantes, Nantes University Hospital, 22 Boulevard Benoni Goullin, 44200 Nantes, France.
Luk H VandenbergheGrousbeck Gene Therapy Center, 20 Staniford Street, Boston, MA 02114, USA; Department of Ophthalmology, Ocular Genomics Institute, Harvard Medical School, 243 Charles Street, Boston, MA 02114, USA; Schepens Eye Research Institute, 20 Staniford Street, Boston MA 02114, USA; Massachusetts Eye and Ear Infirmary, 243 Charles Street, Boston, MA 02114, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA. Electronic address: luk_vandenberghe@meei.harvard.edu.
Nantes Université · FRInserm · FRHarvard University · USMassachusetts Eye and Ear Infirmary · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors are promising clinical candidates for therapeutic gene transfer, and a number of AAV-based drugs may emerge on the market over the coming years. To insure the consistency in efficacy and safety of any drug vial that reaches the patient, regulatory agencies require extensive characterization of the final product. Identity is a key characteristic of a therapeutic product, as it ensures its proper labeling and batch-to-batch consistency. Currently, there is no facile, fast, and robust characterization assay enabling to probe the identity of AAV products at the protein level. Here, we investigated whether the thermostability of AAV particles could inform us on the composition of vector preparations. AAV-ID, an assay based on differential scanning fluorimetry (DSF), was evaluated in two AAV research laboratories for specificity, sensitivity, and reproducibility, for six different serotypes (AAV1, 2, 5, 6.2, 8, and 9), using 67 randomly selected AAV preparations. In addition to enabling discrimination of AAV serotypes based on their melting temperatures, the obtained fluorescent fingerprints also provided information on sample homogeneity, particle concentration, and buffer composition. Our data support the use of AAV-ID as a reproducible, fast, and low-cost method to ensure batch-to-batch consistency in manufacturing facilities and academic laboratories.

Indexed as

DependovirusCapsidCapsid ProteinsGenetic VectorsHumansMutationProtein StabilityReproducibility of ResultsSpectrometry, FluorescenceStructure-Activity RelationshipThermodynamicsCapsid ProteinsAAV vectorsadeno-associated viruscapsid thermostabilityCMCgene therapyhomogeneityidentityidentity assaymanufacturingquality control

Identifiers

PMID28427840
PMCPMC5475248
OpenAlexW2606689543

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.