Evidence map›Paper›PMID 29500218›Full record

ArticleBlood advances2018

Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.

Robert A French, Benjamin J Samelson-Jones, Glenn P Niemeyer, Clinton D Lothrop, Elizabeth P Merricks, Timothy C Nichols, Valder R Arruda

Abstract read
In one paragraph

Article in Blood advances, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

17 citing papers in PubMed.

  1. Article
  2. Article
  3. CRISPR-Mediated In Situ Introduction or Integration ofInternational journal of molecular sciences · 2023
    Article
  4. Article
  5. Worldwide use of factor IX Padua for hemophilia B gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Gene Therapy for Inherited Bleeding Disorders.Seminars in thrombosis and hemostasis · 2021
    Review
  11. AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer.Molecular therapy : the journal of the American Society of Gene Therapy · 2020
    Review
  12. Hemophilia - Impact of Recent Advances on Management.Indian journal of pediatrics · 2020
    Article
  13. Review
  14. Article
  15. Article
  16. Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.Molecular therapy. Methods & clinical development · 2019
    Review
  17. Gene therapy for hemophilia: what does the future hold?Therapeutic advances in hematology · 2018
    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

7 authors.

Robert A FrenchDepartment of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.
Benjamin J Samelson-JonesDepartment of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.ORCID 0000-0001-6772-4140
Glenn P NiemeyerDepartment of Biochemistry, University of Alabama at Birmingham, Birmingham, AL.
Clinton D LothropDepartment of Biochemistry, University of Alabama at Birmingham, Birmingham, AL.
Elizabeth P MerricksDepartment of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC.
Timothy C NicholsDepartment of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC.
Valder R ArrudaDepartment of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.ORCID 0000-0002-8100-5481

Funding

Safety &Efficacy of Intravas. Del. of AAV-F.IX to Skeletal MuscleP01HL064190 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI ARRUDA, VALDER R. · 2000 to 2015
$27.8M
Maintenance of Models of Human Hemophilia and VWDR24HL063098 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI NICHOLS, TIMOTHY CHARLES · 1999 to 2019
$15.1M
Canine Blood Disease Models and Stem Cell ResourceR24HL086944 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI LOTHROP, CLINTON D · 2007 to 2011
$1.7M
NHLBI NIH HHS P01 HL064190NHLBI NIH HHS R24 HL063098NHLBI NIH HHS R24 HL086944
6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

AnimalsDependovirusDisease Models, AnimalDogsFactor IXGenetic TherapyHemophilia BImmune ToleranceMuscle, SkeletalPhenotypeRecombinant ProteinsFactor IXRecombinant Proteins

Identifiers

PMID29500218
PMCPMC5851422

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.