Evidence map›Paper›PMID 35782594›Full record

ArticleMolecular therapy. Methods & clinical development2022

Application of

Su Liu, Lisa Razon, Olivia Ritchie, Choong-Ryoul Sihn, Britta Handyside, Geoffrey Berguig, Jill Woloszynek, Lening Zhang, Paul Batty, David Lillicrap and 8 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.2field-weighted citation impact, top 12% 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

8 citing papers in PubMed, 13 citations in OpenAlex.

  1. Review
  2. Article
  3. iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy.Molecular therapy. Methods & clinical development · 2025
    Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. 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

18 authors at 2 institutions in 2 countries.

Su LiuBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Lisa RazonBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Olivia RitchieBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Choong-Ryoul SihnBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Britta HandysideBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Geoffrey BerguigBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Jill WoloszynekBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Lening ZhangBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Paul BattyDepartment of Pathology and Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, ON K7L 3N6, Canada.
David LillicrapDepartment of Pathology and Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, ON K7L 3N6, Canada.
Vishal AgrawalBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Christa CortesioBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Kahsay GebretsadikBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Hassibullah AkeefeBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Peter ColosiBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Benjamin KimClinical Sciences, BioMarin Pharmaceutical, Inc., Novato, CA 94949, USA.
Stuart BuntingBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Sylvia FongBiology Research, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
BioMarin (United States) · USQueen's University · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recombinant adeno-associated virus (AAV) is an effective platform for therapeutic gene transfer; however, tissue-tropism differences between species are a challenge for successful translation of preclinical results to humans. We evaluated the use of

Indexed as

AAV5-hFVIII-SQAAV transductionAAV vectorshepatocytesin vitro modelvaloctocogene roxaparvovec

Identifiers

PMID35782594
PMCPMC9204658
OpenAlexW4281888846

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.