Evidence map›Paper›PMID 35132205›Full record

ArticleGene therapy2023

Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy.

Carlos Fonck, Cheng Su, Jeremy Arens, Elli Koziol, Jaydeep Srimani, Joshua Henshaw, Andrea Van Tuyl, Sundeep Chandra, Christian Vettermann, Charles A O'Neill

Open access · hybridAbstract read
In one paragraph

Article in Gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
1.7field-weighted citation impact, top 16% 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

6 citing papers in PubMed, 10 citations in OpenAlex.

  1. Trial
  2. A comprehensive atlas of AAV tropism in the mouse.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  3. Gene therapy in hemophilia: the dawn of a new era.Research and practice in thrombosis and haemostasis · 2025
    Review
  4. Review
  5. Valoctocogene Roxaparvovec.Hospital pharmacy · 2024
    Review
  6. 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

10 authors at 1 institution in 1 country.

Carlos Fonck *Pharmacological Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Cheng Su *Data Sciences and Analytics, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Jeremy Arens *Pharmacological Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Elli KoziolPharmacological Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Jaydeep SrimaniClinical Pharmacology, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Joshua HenshawClinical Pharmacology, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Andrea Van TuylBioAnalytical Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Sundeep ChandraPathology, BioMarin Pharmaceutical Inc., Novato, CA, USA.
Christian VettermannBioAnalytical Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA.ORCID 0000-0003-3794-6441
Charles A O'NeillPharmacological Sciences, BioMarin Pharmaceutical Inc., Novato, CA, USA. coneill@bmrn.com.
BioMarin (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype five gene therapy under investigation for the treatment of hemophilia A. Herein, we assessed the potential for germline transmission of AAV5-hFVIII-SQ in mice. Male B6.129S6-Rag2

Indexed as

Factor VIIIGenetic VectorsAdministration, IntravenousAnimalsDependovirusGene Therapy AgentsGenetic TherapyMaleMiceRecombinant Fusion ProteinsFactor VIIIRecombinant Fusion ProteinsValoctocogene Roxaparvovec

Identifiers

PMID35132205
PMCPMC10457182
OpenAlexW4210467854

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.