Evidence map›Paper›PMID 33542328›Full record

ArticleScientific reports2021

Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering.

Nicole L McIntosh, Geoffrey Y Berguig, Omair A Karim, Christa L Cortesio, Rolando De Angelis, Ayesha A Khan, Daniel Gold, John A Maga, Vikas S Bhat

Erratum issuedAbstract read
In one paragraph

Article in Scientific reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 61 papers.

0numbers the graph read from it
0cells of the map it votes in
61citing 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

61 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  5. Review
  6. Barrier Function of the Extracellular Matrix in AAV Gene Therapy.Cellular and molecular bioengineering · 2026
    Article
  7. Article
  8. pH-dependent DNA degradation pathways for adeno-associated virus gene therapy.Molecular therapy. Methods & clinical development · 2025
    Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Characterization of VSV-GP morphology by cryo-EM imaging and SEC-MALS.Molecular therapy. Methods & clinical development · 2025
    Article
  17. Article
  18. Article
  19. Review
  20. Review

1 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors.

Nicole L McIntoshBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Geoffrey Y BerguigBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Omair A KarimBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Christa L CortesioBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Rolando De AngelisBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Ayesha A KhanBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Daniel GoldBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
John A MagaBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA.
Vikas S BhatBioMarin Pharmaceutical, Inc., Novato, CA, 94949, USA. vikas.bhat@bmrn.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno associated virus (AAV) capsids are a leading modality for in vivo gene delivery. Complete and precise characterization of capsid particles, including capsid and vector genome concentration, is necessary to safely and efficaciously dose patients. Size exclusion chromatography (SEC) coupled to multiangle light scattering (MALS) offers a straightforward approach to comprehensively characterize AAV capsids. The current study demonstrates that this method provides detailed AAV characterization information, including but not limited to aggregation profile, size-distribution, capsid content, capsid molar mass, encapsidated DNA molar mass, and total capsid and vector genome titer. Currently, multiple techniques are required to generate this information, with varying accuracy and precision. In the current study, a new series of equations for SEC-MALS are used in tandem with intrinsic properties of the capsids and encapsidated DNA to quantify multiple physical AAV attributes in one 20-min run with minimal sample manipulation, high accuracy, and high precision. These novel applications designate this well-established method as a powerful tool for product development and process analytics in future gene therapy programs.

Identifiers

PMID33542328
PMCPMC7862616

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.