Evidence map›Paper›PMID 34652966›Full record

ArticleHuman gene therapy2022

Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A.

Jenny A Greig, Melanie K Smith, Jayme M L Nordin, Tamara Goode, Edward A Chroscinski, Elizabeth L Buza, Nicole Schmidt, Lisa M Kattenhorn, Samuel Wadsworth, James M Wilson

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 42% 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

1 citing paper in PubMed, 2 citations in OpenAlex.

  1. 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

10 authors at 3 institutions in 2 countries.

Jenny A GreigGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Melanie K SmithGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Jayme M L NordinGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Tamara GoodeGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Edward A ChroscinskiGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Elizabeth L BuzaGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0001-7044-3964
Nicole SchmidtBayer Healthcare Pharmaceuticals, Berlin, Germany.
Lisa M KattenhornUltragenyx Gene Therapy, Cambridge, Massachusetts, USA.
Samuel WadsworthUltragenyx Gene Therapy, Cambridge, Massachusetts, USA.
James M WilsonGene Therapy Program, Perelman School of Medicine, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-9630-3131
University of Pennsylvania · USUltragenyx Pharmaceutical (United States) · USBayer (Germany) · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hemophilia A, a bleeding disorder, affects 1:5,000 males and is caused by a deficiency of human blood coagulation factor VIII (hFVIII). Studies in mice and macaques identified AAVhu37.E03.TTR.hFVIIIco-SQ.PA75 as a clinical candidate gene therapy vector to treat hemophilia A. In this study, we sought to determine the minimally effective dose (MED) of this vector in a hemophilia A mouse model. Mice received one of four vector doses (3 × 10

Indexed as

DependovirusHemophilia AAnimalsDisease Models, AnimalFactor VIIIFemaleGenetic TherapyGenetic VectorsHumansMaleMiceFactor VIIIAAVfactor VIIIgene therapyhemophilia

Identifiers

PMID34652966
PMCPMC9063151
OpenAlexW3207466284

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.